NASA: Space Travel Is Causing Astronauts’ Herpes to Flare Up

Tests show that dormant herpes viruses reactivate in more than half the astronauts who travel on the Space Shuttle and International Space station.

Dormant Viruses

Tests show that dormant herpes viruses reactivate in more than half the astronauts who travel on the Space Shuttle and International Space station, according to new NASA research — a phenomenon the space agency says could pose problems for deep space missions.

“During spaceflight there is a rise in secretion of stress hormones like cortisol and adrenaline, which are known to suppress the immune system, ” said study author Satish Mehta, a researcher at Johnson Space Center, in a press release. “In keeping with this, we find that astronaut’s immune cells — particularly those that normally suppress and eliminate viruses — become less effective during spaceflight and sometimes for up to 60 days after.”

Less Effective

In research published last month in the journal Frontiers in Microbiology, Mehta and colleagues found that astronauts shed more herpes viruses in their urine and saliva than before or after space travel. The culprit, they suspect, is just the stress of spaceflight.

“NASA astronauts endure weeks or even months exposed to microgravity and cosmic radiation — not to mention the extreme G forces of take-off and re-entry,” Mehta said in the press release. “This physical challenge is compounded by more familiar stressors like social separation, confinement and an altered sleep-wake cycle.”

Minor Symptoms

Fortunately, symptoms were relatively rare. Out of 89 astronauts the team studied, only six experienced herpes breakouts in space, according to the paper — a rate of about seven percent.

The viral shedding also got worse the longer the astronauts were off Earth, leading researchers to worry the phenomenon could represent a challenge for deep space travel.

“While only a small proportion develop symptoms, virus reactivation rates increase with spaceflight duration and could present a significant health risk on missions to Mars and beyond,” reads the press release.

READ MORE: Dormant viruses activate during spaceflight [Phys.org]

More on herpes: Immune Cells Working Together To Kill Herpes Virus Captured on Video

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NASA: Space Travel Is Causing Astronauts’ Herpes to Flare Up

Computer Fraud Laws are Flawed, this Lawyer is Fighting Against Them

Tor Ekeland, hacker lawyer, fights back against the harsh punishments decreed using the Computer Fraud and Abuse Act. And one of those fights can be seen in “Trust Machine,” available now at Breaker.io.

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NASA Engineer: Humans Should Consider Settling Saturn’s Moon Titan


Destination: Titan

If the Earth becomes inhabitable, a NASA engineer named Janelle Wellons says we should think about settling Saturn’s moon Titan, and she has a laundry list of reasons why — including that you might be able to fly by flapping your arms.

“It has a thick atmosphere that could help protect us from space radiation,” Wellons wrote on Reddit. “It is so dense that we could actually attach wings to our arms and fly on this moon. I don’t know, it just seems like an awesome place to live.”

Largest Moon

Wellon’s comments came in a Reddit appearance in which NASA engineers, scientists and pilots fielded questions from the public. One Redditor asked where the team would recommend settling if conditions on Earth became untenable, and Wellons chimed in with what she said was a “more interesting answer than the standard Mars or Moon response.”

“How about we consider one of the water worlds in our solar system — Titan,” she wrote. “Titan is the largest moon of Saturn, larger than the planet Mercury even, so I think we could settle with plenty room.”

Swim Good

In spite of Wellon’s enthusiasm, there are definite downsides to Titan. It only gets about one percent of the sunlight Earth does, and according to NASA’s research its maximum temperature is a wintry -292 degrees Fahrenheit.

But Wellon is still a fan.

“Now as for the conditions on the surface — not as rough as you may think,” she wrote. “Titan is the only place besides Earth known to have liquids in the form of lakes and seas on its surface. These liquids are made of methane but, armed with the right kind of protective gear, one could theoretically be able to swim without harm!”

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New Rocket Engine Could Whip You From London to Sydney in 4 Hours

The makers of a new hypersonic rocket engine say it could whip flights from London to Sydney in just four hours, traveling at five times the speed of sound.

Rocket Plane

The makers of a new hypersonic rocket engine say it could whisk flights from London to Sydney in just four hours, traveling at five times the speed of sound. That’s a flight that can take 20 hours on a conventional jetliner.

According to the BBCUK company Reaction Engines says it’s gearing up to test the futuristic craft in Colorado — a startling vision of the future of transportation that could also, if the engine lives up to the hype, inform the future of spaceflight.

Screaming Fist

Reaction Engines, which has backing from the Rolls-Royce and Boeing, calls the new rocket engine the Sabre. It inhales air at lower altitudes, but works more like a rocket when it gets higher up.

“The core can be tested on the ground, but it’s the core that gets you air-breathing from the ground up to the edge of space, at which point there is no more oxygen to breathe and the system transitions to the pure rocket mode,” said Shaun Driscoll, Reaction Engines’ program director, according to the BBC.


The company also says the Sabre engine could push the frontiers of spaceflight, by sending crafts straight into orbit without multiple propellant stages, according to the BBC, which also reported that the the European Space Agency recently signed off on a design review for the Sabre engine.

“The positive conclusion of our Preliminary Design Review marks a major milestone in Sabre development,” ESA’s head of propulsion engineering Mark Ford told the broadcaster. “It confirms the test version of this revolutionary new class of engine is ready for implementation.”

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Astronomers Just Found 83 Giant Black Holes at Universe’s Edge

An international team of researchers says it's found 83 new supermassive black holes at extreme end of the visible universe.

Hole Story

An international team of researchers says it’s found 83 new supermassive black holes at extreme end of the visible universe — by looking at light that took so long to reach Earth that it dates from the early universe.

“It is remarkable that such massive dense objects were able to form so soon after the Big Bang,” said Michael Strauss, a professor of astrophysical sciences at Princeton University involved in the research, in a press release. “Understanding how black holes can form in the early universe, and just how common they are, is a challenge for our cosmological models.”

Squad Goals

The discovery was made by 48 astronomers around the world who described the findings in five new papers in The Astrophysical Journal and the Publications of the Astronomical Observatory of Japan.

The finding was based on data taken with the Hyper Suprime-Cam, a “cutting-edge instrument” at the Subaru Telescope at the National Astronomical Observatory of Japan, in Hawaii, which the researchers combined with readings from three more powerful telescopes around the world.

Quasar Theory

The newly-discovered black holes are quasars, which shoot out matter in powerful jets. The researchers are hoping that more datagathering and analysis will shed light onto how some of the earliest quasars in the universe formed.

“The quasars we discovered will be an interesting subject for further follow-up observations with current and future facilities,” said Yoshiki Matsuok, a researcher at Ehime University who worked on the discovery. “We will also learn about the formation and early evolution of [super massive black holes], by comparing the measured number density and luminosity distribution with predictions from theoretical models.”

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Astronomers Just Found 83 Giant Black Holes at Universe’s Edge

The Pentagon Wants an Orbital Space Weapon to Blast Enemy Missiles

Space Laser

You know the scene in “Akira” where Tetsuo rips a satellite space weapon out of orbit?

Now the U.S. military wants to try something similar, according to Defense One. The Pentagon is requesting hundreds of millions of dollars to ramp up space-based weaponry including particle beams and space lasers that’ll fire downward at Earthly targets — a dark vision of the militarization of space.

Ballistic Missiles

According to Defense One, a Missile Defense Agency document released this week describes the military’s ambitions to disable ballistic missiles right as they launch.

“The addition of the neutral particle Beam effort will design, develop, and conduct a feasibility demonstration for a space-based Directed Energy Intercept layer,” it reads. “This future system will offer new kill options for the [Ballistic Missile Defense System] and adds another layer of protection for the homeland.”

Orbital Weaponry

Several contractors have prepared prototype orbital weaponry designs for the Pentagon over the years, according to Defense One, but they’ve been enormous and impractical. But now officials hope advances could make such a weapon feasible for a test by 2023.

“We’ve come a long way in terms of the technology we use today to where a full, all-up system wouldn’t be the size of three of these conference rooms, right?” said a senior defense official, according to Defense One. “We now believe we can get it down to a package that we can put on as part of a payload to be placed on orbit.”

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Cryptocurrency Price Forecast: Trust Is Growing, But Prices Are Falling

Trust Is Growing…
Before we get to this week’s cryptocurrency news, analysis, and our cryptocurrency price forecast, I want to share an experience from this past week. I was at home watching the NBA playoffs, trying to ignore the commercials, when a strange advertisement caught my eye.

It followed a tomato from its birth on the vine to its end on the dinner table (where it was served as a bolognese sauce), and a diamond from its dusty beginnings to when it sparkled atop an engagement ring.

The voiceover said: “This is a shipment passed 200 times, transparently tracked from port to port. This is the IBM blockchain.”

Let that sink in—IBM.

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Cryptocurrency News: Looking Past the Bithumb Crypto Hack

Another Crypto Hack Derails Recovery
Since our last report, hackers broke into yet another cryptocurrency exchange. This time the target was Bithumb, a Korean exchange known for high-flying prices and ultra-active traders.

While the hackers made off with approximately $31.5 million in funds, the exchange is working with relevant authorities to return the stolen tokens to their respective owners. In the event that some is still missing, the exchange will cover the losses. (Source: “Bithumb Working With Other Crypto Exchanges to Recover Hacked Funds,”.

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Cryptocurrency News: This Week on Bitfinex, Tether, Coinbase, & More

Cryptocurrency News
On the whole, cryptocurrency prices are down from our previous report on cryptos, with the market slipping on news of an exchange being hacked and a report about Bitcoin manipulation.

However, there have been two bright spots: 1) an official from the U.S. Securities and Exchange Commission (SEC) said that Ethereum is not a security, and 2) Coinbase is expanding its selection of tokens.

Let’s start with the good news.
SEC Says ETH Is Not a Security
Investors have some reason to cheer this week. A high-ranking SEC official told attendees of the Yahoo! All Markets Summit: Crypto that Ethereum and Bitcoin are not.

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Cryptocurrency News: This Week on Bitfinex, Tether, Coinbase, & More

Ripple Price Forecast: XRP vs SWIFT, SEC Updates, and More

Ripple vs SWIFT: The War Begins
While most criticisms of XRP do nothing to curb my bullish Ripple price forecast, there is one obstacle that nags at my conscience. Its name is SWIFT.

The Society for Worldwide Interbank Financial Telecommunication (SWIFT) is the king of international payments.

It coordinates wire transfers across 11,000 banks in more than 200 countries and territories, meaning that in order for XRP prices to ascend to $10.00, Ripple needs to launch a successful coup. That is, and always has been, an unwritten part of Ripple’s story.

We’ve seen a lot of progress on that score. In the last three years, Ripple wooed more than 100 financial firms onto its.

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Cryptocurrency News: XRP Validators, Malta, and Practical Tokens

Cryptocurrency News & Market Summary
Investors finally saw some light at the end of the tunnel last week, with cryptos soaring across the board. No one quite knows what kicked off the rally—as it could have been any of the stories we discuss below—but the net result was positive.

Of course, prices won’t stay on this rocket ride forever. I expect to see a resurgence of volatility in short order, because the market is moving as a single unit. Everything is rising in tandem.

This tells me that investors are simply “buying the dip” rather than identifying which cryptos have enough real-world value to outlive the crash.

So if you want to know when.

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Cryptocurrency News: Bitcoin ETFs, Andreessen Horowitz, and Contradictions in Crypto

Cryptocurrency News
This was a bloody week for cryptocurrencies. Everything was covered in red, from Ethereum (ETH) on down to the Basic Attention Token (BAT).

Some investors claim it was inevitable. Others say that price manipulation is to blame.

We think the answers are more complicated than either side has to offer, because our research reveals deep contradictions between the price of cryptos and the underlying development of blockchain projects.

For instance, a leading venture capital (VC) firm launched a $300.0-million crypto investment fund, yet liquidity continues to dry up in crypto markets.

Another example is the U.S. Securities and Exchange Commission’s.

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Cryptocurrency News: New Exchanges Could Boost Crypto Liquidity

Cryptocurrency News
Even though the cryptocurrency news was upbeat in recent days, the market tumbled after the U.S. Securities and Exchange Commission (SEC) rejected calls for a Bitcoin (BTC) exchange-traded fund (ETF).

That news came as a blow to investors, many of whom believe the ETF would open the cryptocurrency industry up to pension funds and other institutional investors. This would create a massive tailwind for cryptos, they say.

So it only follows that a rejection of the Bitcoin ETF should send cryptos tumbling, correct? Well, maybe you can follow that logic. To me, it seems like a dramatic overreaction.

I understand that legitimizing cryptos is important. But.

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Cryptocurrency News: Bitcoin ETF Rejection, AMD Microchip Sales, and Hedge Funds

Cryptocurrency News
Although cryptocurrency prices were heating up last week (Bitcoin, especially), regulators poured cold water on the rally by rejecting calls for a Bitcoin exchange-traded fund (ETF). This is the second time that the proposal fell on deaf ears. (More on that below.)

Crypto mining ran into similar trouble, as you can see from Advanced Micro Devices, Inc.‘s (NASDAQ:AMD) most recent quarterly earnings. However, it wasn’t all bad news. Investors should, for instance, be cheering the fact that hedge funds are ramping up their involvement in cryptocurrency markets.

Without further ado, here are those stories in greater detail.
ETF Rejection.

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Cryptocurrency News: What You Need to Know This Week

Cryptocurrency News
Cryptocurrencies traded sideways since our last report on cryptos. However, I noticed something interesting when playing around with Yahoo! Finance’s cryptocurrency screener: There are profitable pockets in this market.

Incidentally, Yahoo’s screener is far superior to the one on CoinMarketCap, so if you’re looking to compare digital assets, I highly recommend it.

But let’s get back to my epiphany.

In the last month, at one point or another, most crypto assets on our favorites list saw double-digit increases. It’s true that each upswing was followed by a hard crash, but investors who rode the trend would have made a.

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Cryptocurrency News: Vitalik Buterin Doesn’t Care About Bitcoin ETFs

Cryptocurrency News
While headline numbers look devastating this week, investors might take some solace in knowing that cryptocurrencies found their bottom at roughly $189.8 billion in market cap—that was the low point. Since then, investors put more than $20.0 billion back into the market.

During the rout, Ethereum broke below $300.00 and XRP fell below $0.30, marking yearly lows for both tokens. The same was true down the list of the top 100 biggest cryptos.

Altcoins took the brunt of the hit. BTC Dominance, which reveals how tightly investment is concentrated in Bitcoin, rose from 42.62% to 53.27% in just one month, showing that investors either fled altcoins at higher.

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Gene therapy – Wikipedia

In the medicine field gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat disease.[1][2] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.[3] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.

Between 1989 and February 2016, over 2,300 clinical trials were conducted, with more than half of them in phase I.[4]

Not all medical procedures that introduce alterations to a patient’s genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[5] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect.

Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.

The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980.[6][7] Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified[8] and even if he is correct, it’s unlikely it produced any significant beneficial effects treating beta-thalassemia.

After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA-SCID.[9]

The first somatic treatment that produced a permanent genetic change was initiated in 1993. The goal was to cure malignant brain tumors by using recombinant DNA to transfer a gene making the tumor cells sensitive to a drug that in turn would cause the tumor cells to die.[10]

Gene therapy is a way to fix a genetic problem at its source. The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations.

The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a “vector”, which carries the molecule inside cells.

Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers’ attention, although as of 2014[update], it was still largely an experimental technique.[11] These include treatment of retinal diseases Leber’s congenital amaurosis[12][13][14][15] and choroideremia,[16] X-linked SCID,[17] ADA-SCID,[18][19] adrenoleukodystrophy,[20] chronic lymphocytic leukemia (CLL),[21] acute lymphocytic leukemia (ALL),[22] multiple myeloma,[23] haemophilia,[19] and Parkinson’s disease.[24] Between 2013 and April 2014, US companies invested over $600 million in the field.[25]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[26] In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.[27]In 2012 Glybera, a treatment for a rare inherited disorder, Lipoprotein lipase deficiency became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[11][28]

Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered replacing or disrupting defective genes.[29] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[28]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[30] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[31][32] Naked DNA approaches have also been explored, especially in the context of vaccine development.[33]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014[update] these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[34]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[35] viral diseases,[36] and cancer.[37] As of 2016[update] these approaches were still years from being medicine.[38][39]

Gene therapy may be classified into two types:

In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell. Any such modifications affect the individual patient only, and are not inherited by offspring. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.

Over 600 clinical trials utilizing SCGT are underway[when?] in the US. Most focus on severe genetic disorders, including immunodeficiencies, haemophilia, thalassaemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy. The complete correction of a genetic disorder or the replacement of multiple genes is not yet possible. Only a few of the trials are in the advanced stages.[40] [needs update]

In germline gene therapy (GGT), germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes. Modifying a germ cell causes all the organism’s cells to contain the modified gene. The change is therefore heritable and passed on to later generations. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[41] prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[41] and higher risks versus SCGT.[42] The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).[41][43][44][45]

The delivery of DNA into cells can be accomplished by multiple methods. The two major classes are recombinant viruses (sometimes called biological nanoparticles or viral vectors) and naked DNA or DNA complexes (non-viral methods).

In order to replicate, viruses introduce their genetic material into the host cell, tricking the host’s cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell. Scientists exploit this by substituting a virus’s genetic material with therapeutic DNA. (The term ‘DNA’ may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well.) A number of viruses have been used for human gene therapy, including retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus.[4] Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host’s genome, becoming a permanent part of the host’s DNA in infected cells.

Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Later technology remedied this deficiency.[citation needed]

Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.

Some of the unsolved problems include:

Three patients’ deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger, who died in 1999 because of immune rejection response.[52] One X-SCID patient died of leukemia in 2003.[9] In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy.[53]

In 1972 Friedmann and Roblin authored a paper in Science titled “Gene therapy for human genetic disease?”[54] Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects.[55]

In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.[56]

The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.[57] Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient’s blood cells was replaced by the functional variant. Ashantis immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary.[58]

Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).[59] The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocolno.1602 November 24, 1993,[60] and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena.

In 1992 Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.[61] In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID). The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or “bubble boy” disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial’s Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany.[62]

In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. Blood was removed from his mother’s placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase (ADA) was obtained and inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew’s blood. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.[63]

Jesse Gelsinger’s death in 1999 impeded gene therapy research in the US.[64][65] As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.[66]

The modified cancer gene therapy strategy of antisense IGF-I RNA (NIH n 1602)[60] using antisense / triple helix anti-IGF-I approach was registered in 2002 by Wiley gene therapy clinical trial – n 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening anti-tumor immune and apoptotic phenomena.

Sickle-cell disease can be treated in mice.[67] The mice which have essentially the same defect that causes human cases used a viral vector to induce production of fetal hemoglobin (HbF), which normally ceases to be produced shortly after birth. In humans, the use of hydroxyurea to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production.[68]

A new gene therapy approach repaired errors in messenger RNA derived from defective genes. This technique has the potential to treat thalassaemia, cystic fibrosis and some cancers.[69]

Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane.[70]

In 2003 a research team inserted genes into the brain for the first time. They used liposomes coated in a polymer called polyethylene glycol, which unlike viral vectors, are small enough to cross the bloodbrain barrier.[71]

Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.[72]

Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus. In 2003, it was approved in China for the treatment of head and neck squamous cell carcinoma.[26]

In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system.[73]

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene.[74] Similar to organ transplantation, gene therapy has been plagued by this problem. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as microRNAs. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene.

In August scientists successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells.[75]

In November researchers reported on the use of VRX496, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial, five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. A single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial.[76][77]

In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007.[78]

Leber’s congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April.[12] Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects.[12][13][14][15]

In September researchers were able to give trichromatic vision to squirrel monkeys.[79] In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.[80]

An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two young specimens. The therapy was less efficient for older dogs.[81]

In September it was announced that an 18-year-old male patient in France with beta-thalassemia major had been successfully treated.[82] Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions.[83] The technique used a lentiviral vector to transduce the human -globin gene into purified blood and marrow cells obtained from the patient in June 2007.[84] The patient’s haemoglobin levels were stable at 9 to 10 g/dL. About a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions were not needed.[84][85] Further clinical trials were planned.[86] Bone marrow transplants are the only cure for thalassemia, but 75% of patients do not find a matching donor.[85]

Cancer immunogene therapy using modified antigene, antisense/triple helix approach was introduced in South America in 2010/11 in La Sabana University, Bogota (Ethical Committee 14 December 2010, no P-004-10). Considering the ethical aspect of gene diagnostic and gene therapy targeting IGF-I, the IGF-I expressing tumors i.e. lung and epidermis cancers were treated (Trojan et al. 2016).[87][88]

In 2007 and 2008, a man (Timothy Ray Brown) was cured of HIV by repeated hematopoietic stem cell transplantation (see also allogeneic stem cell transplantation, allogeneic bone marrow transplantation, allotransplantation) with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011.[89] It required complete ablation of existing bone marrow, which is very debilitating.

In August two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease.[21] In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.[90]

Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction.[91][92]

In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF.[93][27] Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF.[94][95]

The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for 10 participants in July.[96] The study was expected to continue until 2015.[86]

In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis.[97] The recommendation was endorsed by the European Commission in November 2012[11][28][98][99] and commercial rollout began in late 2014.[100] Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,[101] revised to $1 million in 2015,[102] making it the most expensive medicine in the world at the time.[103] As of 2016[update], only the patients treated in clinical trials and a patient who paid the full price for treatment have received the drug.[104]

In December 2012, it was reported that 10 of 13 patients with multiple myeloma were in remission “or very close to it” three months after being injected with a treatment involving genetically engineered T cells to target proteins NY-ESO-1 and LAGE-1, which exist only on cancerous myeloma cells.[23]

In March researchers reported that three of five adult subjects who had acute lymphocytic leukemia (ALL) had been in remission for five months to two years after being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients’ immune systems would make normal T-cells and B-cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease.[22]

Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients[105] at several hospitals to combat heart disease. The therapy was designed to increase the levels of SERCA2, a protein in heart muscles, improving muscle function.[106] The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process.[107] In 2016 it was reported that no improvement was found from the CUPID 2 trial.[108]

In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 732 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills.[109] The other children had Wiskott-Aldrich syndrome, which leaves them to open to infection, autoimmune diseases, and cancer.[110] Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising.[111][112]

In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress.[19] In 2014 a further 18 children with ADA-SCID were cured by gene therapy.[113] ADA-SCID children have no functioning immune system and are sometimes known as “bubble children.”[19]

Also in October researchers reported that they had treated six hemophilia sufferers in early 2011 using an adeno-associated virus. Over two years later all six were producing clotting factor.[19][114]

In January researchers reported that six choroideremia patients had been treated with adeno-associated virus with a copy of REP1. Over a six-month to two-year period all had improved their sight.[115][116] By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.[16] Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight.

In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results.[117][118]

Clinical trials of gene therapy for sickle cell disease were started in 2014.[119][120] There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease.[121][needs update]

In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA “breakthrough” status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease.[122]

In March researchers delivered a recombinant gene encoding a broadly neutralizing antibody into monkeys infected with simian HIV; the monkeys’ cells produced the antibody, which cleared them of HIV. The technique is named immunoprophylaxis by gene transfer (IGT). Animal tests for antibodies to ebola, malaria, influenza, and hepatitis were underway.[123][124]

In March, scientists, including an inventor of CRISPR, Jennifer Doudna, urged a worldwide moratorium on germline gene therapy, writing “scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans” until the full implications “are discussed among scientific and governmental organizations”.[125][126][127][128]

In October, researchers announced that they had treated a baby girl, Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]).[129] Children with highly aggressive ALL normally have a very poor prognosis and Layla’s disease had been regarded as terminal before the treatment.[130]

In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies[131] but that basic research including embryo gene editing should continue.[132]

In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis[133][134] and the European Commission approved it in June.[135] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe.[136]

In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the immune response) removed using CRISPR-Cas9.[137][138]

A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy.[139]

In February Kite Pharma announced results from a clinical trial of CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma.[140]

In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease.[141]

In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia.[142] Tisagenlecleucel is an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia; T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor (a chimeric T cell receptor, or “CAR-T”) that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. This is the first form of gene therapy to be approved in the United States. In October, a similar therapy called axicabtagene ciloleucel was approved for non-Hodgkin lymphoma.[143]

In December the results of using an adeno-associated virus with blood clotting factor VIII to treat nine haemophilia A patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels. The low and medium dose regimes had no effect on the patient’s blood clotting levels.[144][145]

In December, the FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber’s congenital amaurosis.[146] The price of this treatment was 850,000 US dollars for both eyes.[147][148]

In February 2019, medical scientists working with Sangamo Therapeutics, headquartered in Richmond, California, announced the first ever “in body” human gene editing therapy to permanently alter DNA – in a patient with Hunter Syndrome.[149] Clinical trials by Sangamo involving gene editing using Zinc Finger Nuclease (ZFN) are ongoing.[150]

Speculated uses for gene therapy include:

Athletes might adopt gene therapy technologies to improve their performance.[151] Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.[152]

Genetic engineering could be used to cure diseases, but also to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. Ethical claims about germline engineering include beliefs that every fetus has a right to remain genetically unmodified, that parents hold the right to genetically modify their offspring, and that every child has the right to be born free of preventable diseases.[153][154][155] For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.[156][157] Another theorist claims that moral concerns limit but do not prohibit germline engineering.[158]

Possible regulatory schemes include a complete ban, provision to everyone, or professional self-regulation. The American Medical Associations Council on Ethical and Judicial Affairs stated that “genetic interventions to enhance traits should be considered permissible only in severely restricted situations: (1) clear and meaningful benefits to the fetus or child; (2) no trade-off with other characteristics or traits; and (3) equal access to the genetic technology, irrespective of income or other socioeconomic characteristics.”[159]

As early in the history of biotechnology as 1990, there have been scientists opposed to attempts to modify the human germline using these new tools,[160] and such concerns have continued as technology progressed.[161][162] With the advent of new techniques like CRISPR, in March 2015 a group of scientists urged a worldwide moratorium on clinical use of gene editing technologies to edit the human genome in a way that can be inherited.[125][126][127][128] In April 2015, researchers sparked controversy when they reported results of basic research to edit the DNA of non-viable human embryos using CRISPR.[163][164] A committee of the American National Academy of Sciences and National Academy of Medicine gave qualified support to human genome editing in 2017[165][166] once answers have been found to safety and efficiency problems “but only for serious conditions under stringent oversight.”[167]

Regulations covering genetic modification are part of general guidelines about human-involved biomedical research. There are no international treaties which are legally binding in this area, but there are recommendations for national laws from various bodies.

The Helsinki Declaration (Ethical Principles for Medical Research Involving Human Subjects) was amended by the World Medical Association’s General Assembly in 2008. This document provides principles physicians and researchers must consider when involving humans as research subjects. The Statement on Gene Therapy Research initiated by the Human Genome Organization (HUGO) in 2001 provides a legal baseline for all countries. HUGOs document emphasizes human freedom and adherence to human rights, and offers recommendations for somatic gene therapy, including the importance of recognizing public concerns about such research.[168]

No federal legislation lays out protocols or restrictions about human genetic engineering. This subject is governed by overlapping regulations from local and federal agencies, including the Department of Health and Human Services, the FDA and NIH’s Recombinant DNA Advisory Committee. Researchers seeking federal funds for an investigational new drug application, (commonly the case for somatic human genetic engineering,) must obey international and federal guidelines for the protection of human subjects.[169]

NIH serves as the main gene therapy regulator for federally funded research. Privately funded research is advised to follow these regulations. NIH provides funding for research that develops or enhances genetic engineering techniques and to evaluate the ethics and quality in current research. The NIH maintains a mandatory registry of human genetic engineering research protocols that includes all federally funded projects.

An NIH advisory committee published a set of guidelines on gene manipulation.[170] The guidelines discuss lab safety as well as human test subjects and various experimental types that involve genetic changes. Several sections specifically pertain to human genetic engineering, including Section III-C-1. This section describes required review processes and other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient.[171] The protocol for a gene therapy clinical trial must be approved by the NIH’s Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial.[170]

As with other kinds of drugs, the FDA regulates the quality and safety of gene therapy products and supervises how these products are used clinically. Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects, such as clinical trials, must be reviewed and approved by the FDA and an Institutional Review Board.[172][173]

Gene therapy is the basis for the plotline of the film I Am Legend[174] and the TV show Will Gene Therapy Change the Human Race?.[175] In 1994, gene therapy was a plot element in “The Erlenmeyer Flask”, the first season finale of The X-Files; it is also used in Stargate as a means of allowing humans to use Ancient technology.[176][177]

See the article here:

Gene therapy – Wikipedia

Gene Therapy – Sickle Cell Anemia News

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.


Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy – Sickle Cell Anemia News

What is gene therapy? – Genetics Home Reference – NIH

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.


What is gene therapy? – Genetics Home Reference – NIH

Boeing Is Prepping to Launch Astronauts to Space Station

Commercial Crew Program

SpaceX isn’t the only company attempting to revolutionize the way we send astronauts to space.

Boeing, the largest aerospace company in the world, is looking to send up its own take on a passenger spacecraft, which it calls the CST-100 Starliner, to the International Space Station. Boeing is planning to launch the capsule — uncrewed for now, as a test flight — on an Atlas 5 rocket as early as April, according to NASA.


Boeing’s commercial spacecraft shares similarities with SpaceX’s Crew Dragon: it can seat a crew of seven, be operated from a central control panel, dock autonomously with the ISS, and can also be reused multiple times.

Boeing’s Starliner is the result of a $4.2 billion contract signed with NASA in 2014 under the Commercial Crew Program. SpaceX signed a very similar contract for its Crew Dragon mission at the same time, although it paid SpaceX just $2.4 billion.

Race to the ISS

SpaceX successfully launched its passenger spacecraft to the ISS on Saturday, becoming the first ever private American spacecraft to do so. It also marked the first time astronauts launched into space from American soil since the end of NASA’s Space Shuttle program in 2011.

Boeing has tests to complete before it takes off.

“There still are many critical steps to complete before launch and while we eagerly are anticipating these launches, we will step through our test flight preparations and readiness reviews,” Kathy Lueders, Commercial Crew Program manager at NASA said in an official update.

SpaceX is planning a crewed test flight in July of this year. Boeing wants to do the same only a month later — and its first pilots are already on stand-by.

READ MORE: Crew Dragon and Starliner: A Look at the Upcoming Astronaut Taxis [Space.com]

More on Starliner: NASA Announces The First Commercial Astronauts to Pilot The Next Generation of Spacecraft

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Boeing Is Prepping to Launch Astronauts to Space Station