UCLA Research Shows Promising Method For Correcting Genetic Code To Treat Sickle Cell Disease

Posted: Thursday, March 12, 2015 7:08 PM

UCLA stem-cell researchers have shown that a novel stem-cell gene therapy method could one day provide a one-time, lasting treatment for the most common inherited blood disorder in the U.S. sickle cell disease. Publishedin the journal Blood, the study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, the gene correction method leads to the production of normal red blood cells. The study was directed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member, Dr. Donald Kohn.

People with sickle cell disease are born with a mutation in their beta-globin gene, which is responsible for delivering oxygen to the body through blood circulation. The mutation causes blood stem cellswhich are made in the bone marrowto produce distorted and rigid red blood cells that resemble a crescent or sickle shape. Consequently, the abnormally shaped red blood cells do not move smoothly through blood vessels, resulting in insufficient oxygen supply to vital organs. Anyone can be born with sickle cell disease, but it occurs more frequently in African Americans and Hispanic Americans.

The stem-cell gene therapy method described in the study seeks to directly correct the mutation in the beta-globin gene so bone marrow stem cells then produce normal, circular-shaped blood cells that do not sickle. The fascinating gene correction technique used specially engineered enzymes, called zinc-finger nucleases, tocut out the mutated genetic code and replace it with a corrected version that repairs the beta-globin mutation.

For the study, bone marrow stem cells donated by people with the sickle cell gene mutation were treated in the laboratory with the zinc-finger nucleases enzyme cutting method.Kohn and his team then demonstrated in mouse models that thecorrected bone-marrow stem cells have the capability to replicate successfully. The research showed that the method holds the potential to permanently treat the disease if a higher level of correction is achieved.

This is a very exciting result,said Dr. Kohn, professor of pediatrics atUCLAs David Geffen School of Medicine, professor of microbiology, immunology and molecular genetics in Life Sciences at UCLA, member of the UCLA Childrens Discovery and Innovation Institute at Mattel Childrens Hospital and senior author on the study. It suggests the future direction for treating genetic diseases will be by correcting the specific mutation in a patients genetic code. Since sickle cell disease was the first human genetic disease where we understood the fundamental gene defect,and since everyone with sickle cell has the exact same mutation in the beta-globin gene, it is a great target for this gene correction method.

To make the cut in the genetic code, Dr. Kohn and his team used zinc-finger nucleases engineered by Sangamo BioSciences, Inc., in Richmond. The enzymes can be designed to recognize a specific and targeted point in the genetic code. For the study, scientists at Sangamo BioSciences engineered the enzymes to create a cut at the site of the mutated genetic code that causes sickle cell disease. This break triggered a natural process of repair in the cell and at the same time, a molecule containing the correct genetic code was inserted to replace the mutated code.

The next steps in this research will involve improving the efficiency of the mutation correction process and performing pre-clinical studies to demonstrate that the method is effective and safe enough to move to clinical trials.

Symptoms of sickle cell disease usually begin in early childhood and include a low number of red blood cells (anemia), repeated infections and periodic episodes of pain. People with sickle cell disease typically have a shortened lifespan of just 36-40 years of age. The disease impacts more than 250,000 new patients worldwide each year. The only cure currently available for sickle cell disease is a transplant of bone marrow stem cells from a matched sibling, but matches are rare or can result in rejection of the transplanted cells.

This is a promising first step in showing that gene correction has the potential to help patients with sickle cell disease, said Megan Hoban, a senior graduate student in microbiology, immunology and molecular genetics and first author on the study. The study data provide the foundational evidence that the method is viable.

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UCLA Research Shows Promising Method For Correcting Genetic Code To Treat Sickle Cell Disease

Why NASA looks to Europa to find the building blocks of life

NASA wants to send an expedition to Jupiter’s moon, Europa, to find the ocean it believes lies under the ice — and the building blocks for life it thinks it will find there.

Screenshot by Michelle Starr/CNET

When it comes to life, biologists have long hypothesised that its origins — on Earth, at least — were in thermal vents on the ocean floor, following a period of spontaneous metabolism before life began. Thermal vents are rich sites for marine life — especially in Antarctica where, in the darkness under the ice, creatures proliferate in the warm, mineral-rich waters streaming from the vents.

It’s just one of several theories, but if it’s correct, Jupiter’s moon Europa could be a very exciting place indeed. Ever since plumes of vapour were discovered on Jupiter’s moon in December last year, NASA has been floating theories about the oceans that may be sloshing away under the layer of surface ice — its similarities to theories about the early Earth look like it may have the ingredients for life.

“[There is] an ocean in our solar system that has been in existence for billions of years. It’s an ocean that is perhaps ten times as deep as Earth’s ocean. It’s an ocean that is global and may contain two to three times the volume of all the liquid water on Earth. It’s an ocean that exists beneath the icy shell of Jupiter’s moon, Europa,” said NASA astrobiologist John Hand in a new NASA video.

In its early days, before the formation of the ozone layer, Earth’s atmosphere was largely devoid of oxygen, and we know that the Earth has been all but covered in a layer of ice in several ice ages past. These ice layers, which provided protection from harsh ultraviolet light and a crude atmosphere, combined with thermal vents — sites of propagation for single-celled organisms — could very well bear strong similarities to Europa, with its layer of ice and crude atmosphere.

Courtesy Chris German, WHOI/NSF, NASA/ROV

Scientists at NASA’s Jet Propulsion Laboratory are studying the life around these thermal vents on Earth in order to collect clues about what life on Europa might look like. In particular, they’re looking at a type of shrimp called Rimicaris hybisae, living on one of the world’s deepest hydrothermal vents, in the Caribbean Sea.

“For two-thirds of the Earth’s history, life has existed only as microbial life,” said JPL senior research scientist Max Coleman. “On Europa, the best chance for life would be microbial.”

Life on thermal vents is able to survive extraordinarily harsh conditions; bacteria, for instance, survives without sunlight — and therefore photosynthesis — by relying instead on chemosynthesis: a process whereby organisms obtain energy from chemical reactions. In the case of bacteria on the hydrothermal vent, the bacteria use hydrogen sulphide produced by the vents to produce organic matter. Although hydrogen sulphide is toxic to organisms in high concentrations, the bacteria have adapted by positioning themselves directly between the sulphide-rich water and the normal ocean water.

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Why NASA looks to Europa to find the building blocks of life

Space station gets zero-gravity 3-D printer

WASHINGTON, Nov. 18 (UPI) — The International Space Station is now home to a 3-D printer, after NASA astronaut Barry Wilmore spent most of Monday unpacking and installing the machine. He and his colleagues will soon begin experimenting with additive manufacturing in microgravity.

The printer was engineered by the California-based company Made In Space and delivered to the space station in late September, but its installation had to be put off while astronauts wrapped up a few time-sensitive experiments.

If the new printer works properly in space, it could help ISS astronauts become more self-sufficient — allowing them to design and print their own tools and gadgets right on board instead of waiting for another resupply cargo ship to be launched from Earth.

To ensure the printer is unaffected by microgravity, astronauts will begin by printing a pair of “engineering coupons.” The coupons will be sent back to mission control where they’ll be compared to coupons that were printed before the machine was packaged and shipped into outer space.

“This is a very exciting day for me and the rest of the team. We had to conquer many technical challenges to get the 3D printer to this stage,” Made In Space lead engineer Mike Snyder said in a company press release. “This experiment has been an advantageous first stepping stone to the future ability to manufacture a large portion of materials and equipment in space that has been traditionally launched from Earth surface, which will completely change our methods of exploration.”

Snyder and his colleagues are confident in their work and expect the machine to perform as advertised.

“We have really high expectations for it printing,” Jason Dunn, the company’s chief technology officer, said in an interview earlier this year. “We’ve done all the zero gravity research we could on the airplane. (But) there’s always the things we can’t test that you can only do once you’re up there.”

2014 United Press International, Inc. All Rights Reserved. Any reproduction, republication, redistribution and/or modification of any UPI content is expressly prohibited without UPI’s prior written consent.

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Space station gets zero-gravity 3-D printer

Two families of comets found around nearby star

Beta Pictoris is a young star located about 63 light-years from the Sun. It is only about 20 million years old and is surrounded by a huge disc of material – a very active young planetary system where gas and dust are produced by the evaporation of comets and the collisions of asteroids.

Flavien Kiefer (IAP/CNRS/UPMC), lead author of the new study sets the scene: “Beta Pictoris is a very exciting target! The detailed observations of its exocomets give us clues to help understand what processes occur in this kind of young planetary system.”

For almost 30 years astronomers have seen subtle changes in the light from Beta Pictoris that were thought to be caused by the passage of comets in front of the star itself. Comets are small bodies of a few kilometres in size, but they are rich in ices, which evaporate when they approach their star, producing gigantic tails of gas and dust that can absorb some of the light passing through them.

The dim light from the exocomets is swamped by the light of the brilliant star so they cannot be imaged directly from Earth.

To study the Beta Pictoris exocomets, the team analysed more than 1000 observations obtained between 2003 and 2011 with the HARPS instrument on the ESO 3.6-metre telescope at the La Silla Observatory in Chile.

The researchers selected a sample of 493 different exocomets. Some exocomets were observed several times and for a few hours. Careful analysis provided measurements of the speed and the size of the gas clouds. Some of the orbital properties of each of these exocomets, such as the shape and the orientation of the orbit and the distance to the star, could also be deduced.

This analysis of several hundreds of exocomets in a single exo-planetary system is unique.

It revealed the presence of two distinct families of exocomets: one family of old exocomets whose orbits are controlled by a massive planet [1], and another family, probably arising from the recent breakdown of one or a few bigger objects. Different families of comets also exist in the Solar System.

The exocomets of the first family have a variety of orbits and show a rather weak activity with low production rates of gas and dust. This suggests that these comets have exhausted their supplies of ices during their multiple passages close to Beta Pictoris [2].

The exocomets of the second family are much more active and are also on nearly identical orbits [3]. This suggests that the members of the second family all arise from the same origin: probably the breakdown of a larger object whose fragments are on an orbit grazing the star Beta Pictoris.

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Two families of comets found around nearby star

ROWING | Red Competes at 50th Head of Charles Regatta

By TUCKER MAGGIO-HUCEK

The Cornell mens lightweight, heavyweight and womens rowing teams kicked off their fall season in Cambridge, Massachusetts, this past weekend. All three teams competed in the 50th Head of the Charles Regatta that had over 2,200 entries from all different backgrounds.

The mens lightweight team had two boats race on Sunday. The lightweight eights boat, which was led by senior captain Chris Rogers, finished the race in third place behind two Ivy League rivals. The race was extremely competitive, with all three boats finishing within .05 seconds of each other, as Princeton and Harvard edged out Cornell for first and second, respectively.

The eight had a really tight race and put us in a great position for the rest of the season and for winter and spring training, said senior captain Richard Richhart.

The other lightweight boat competed in the fours and had a strong showing, finishing in third behind two international foes. The London Training Center, who won the race, bested the Cornell boat by a large margin but junior Ian Sigal led the boat ahead of all other collegiate entries.

The four truly defined themselves against our League competition. They placed third and were only bested by international competition. Were all really proud of those athletes, Richhart said.

The heavyweights had two boats take to the waters in Massachusetts. In the mens championship fours the Red took seventh place and finished under 40 seconds behind the winners from Camp Randall, a rowing club from Wisconsin. The University of Wisconsin finished in sixth place, finishing just over three seconds before the Cornell boat.

In the mens championship eights, the heavyweight boat had a rough outing and finishing in 33rd out of 36 boats, thanks to a pair of collisions and a one minute penalty that was added on to their time. Harvard, Princeton and Yale all placed within the top-ten.

Despite being forced into some difficult situations, the womens boats started their fall season off in fine fashion. In the womens championship eights, the Cornell boat finished in 11th place in a race that included the United States National Team, Cambridge and Oxford.

The varsity eight had a very promising performance. We nearly collided with several boats and had a very exciting race, said senior captain Elyce Buell. Despite this we had a very competitive time compared to the other crews.

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ROWING | Red Competes at 50th Head of Charles Regatta

Two families of comets found around nearby star: Biggest census ever of exocomets around beta pictoris

The HARPS instrument at ESO’s La Silla Observatory in Chile has been used to make the most complete census of comets around another star ever created. A French team of astronomers has studied nearly 500 individual comets orbiting the star Beta Pictoris and has discovered that they belong to two distinct families of exocomets: old exocomets that have made multiple passages near the star, and younger exocomets that probably came from the recent breakup of one or more larger objects. The new results will appear in the journal Nature on 23 October 2014.

Beta Pictoris is a young star located about 63 light-years from the Sun. It is only about 20 million years old and is surrounded by a huge disc of material — a very active young planetary system where gas and dust are produced by the evaporation of comets and the collisions of asteroids.

Flavien Kiefer (IAP/CNRS/UPMC), lead author of the new study sets the scene: “Beta Pictoris is a very exciting target! The detailed observations of its exocomets give us clues to help understand what processes occur in this kind of young planetary system.”

For almost 30 years astronomers have seen subtle changes in the light from Beta Pictoris that were thought to be caused by the passage of comets in front of the star itself. Comets are small bodies of a few kilometres in size, but they are rich in ices, which evaporate when they approach their star, producing gigantic tails of gas and dust that can absorb some of the light passing through them. The dim light from the exocomets is swamped by the light of the brilliant star so they cannot be imaged directly from Earth.

To study the Beta Pictoris exocomets, the team analysed more than 1000 observations obtained between 2003 and 2011 with the HARPS instrument on the ESO 3.6-metre telescope at the La Silla Observatory in Chile.

The researchers selected a sample of 493 different exocomets. Some exocomets were observed several times and for a few hours. Careful analysis provided measurements of the speed and the size of the gas clouds. Some of the orbital properties of each of these exocomets, such as the shape and the orientation of the orbit and the distance to the star, could also be deduced.

This analysis of several hundreds of exocomets in a single exo-planetary system is unique. It revealed the presence of two distinct families of exocomets: one family of old exocomets whose orbits are controlled by a massive planet [1], and another family, probably arising from the recent breakdown of one or a few bigger objects. Different families of comets also exist in the Solar System.

The exocomets of the first family have a variety of orbits and show a rather weak activity with low production rates of gas and dust. This suggests that these comets have exhausted their supplies of ices during their multiple passages close to Beta Pictoris [2].

The exocomets of the second family are much more active and are also on nearly identical orbits [3]. This suggests that the members of the second family all arise from the same origin: probably the breakdown of a larger object whose fragments are on an orbit grazing the star Beta Pictoris.

Flavien Kiefer concludes: “For the first time a statistical study has determined the physics and orbits for a large number of exocomets. This work provides a remarkable look at the mechanisms that were at work in the Solar System just after its formation 4.5 billion years ago.”

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Two families of comets found around nearby star: Biggest census ever of exocomets around beta pictoris

Penn Medicine University City Unveiled: Market Street Tower is Primed to Boost Patient Engagement, Elevate the …

PHILADELPHIA The dedication of Penn Medicine University City on September 12 will mark the grand opening of Penn Medicines newest outpatient facility, which showcases a new model for patient experience and engagement. The facility was developed with a $38 million investment from Penn Medicine, and in partnership with Good Shepherd Penn Partners, The University City Science Center and Wexford Science & Technology. The 150,000 square-foot outpatient site is now home to more than a dozen clinical specialties whose staff work together in multi-disciplinary teams aided by state-of-the-art medical technology to provide both a modern, one-stop-shop clinic for outpatient services, as well as an ambulatory surgical center and a 29,800-square-foot therapy and rehabilitation facility.

With nearly 110 exam rooms, six outpatient operating rooms, and an outpatient medical imaging and diagnostic testing center, Penn Medicine University City serves as Penn Presbyterian Medical Centers most comprehensive ambulatory care site. Its location expands Penns footprint within the West Philadelphia community, bringing more health care services closer to where people work. The facility also serves as a new front door to Penns campus from University City onto the growing Market Street medical and Science Center corridor, providing easier access for patients coming from West Philadelphia and the nearby suburbs.

This is a very exciting time to be at Penn Presbyterian Medical Center, said Michele Volpe, executive director of Penn Presbyterian. With University Citys opening, we celebrate the first of many upcoming milestones for PPMC. The lessons learned throughout the design and development process of this extraordinary facility will inform the work we do each day in our existing facilities, helping us to continue building upon our already rich culture of excellence and map new strategies to deliver the very best patient care.

Penn Medicine University City serves as the first of many firsts for Penn Presbyterian, and the beginning of a major expansion for Penn Medicine. With the completion of the new Pavilion for Advanced Care scheduled for early 2015 the Health Systems regional Level I Trauma and Critical Care Center will transition from its current home at HUP, making way for a new patient facility.

Penn Medicine University City is an integral part of a series of development projects for our health system, said Ralph Muller, CEO of the University of Pennsylvania Health System. Each phase is designed to provide patients with the exceptional care for which Penn Medicine is known, throughout the City of Philadelphia and the region.

A preeminent example of Penn Medicine University Citys new patient engagement model is the Penn Musculoskeletal Center, the first enterprise of its kind in the region. The center offers a unique and highly specialized approach to treating orthopaedic disorders, injuries and other conditions of the joints, bones or muscles. On-site clinical specialties include orthopaedic surgery, rheumatology, physical medicine and rehabilitation, internal medicine, pain medicine, and therapy/rehabilitation services.The center will also be home to the new Penn Center for Human Performance, slated to open later this year.

Were offering a whole new approach to integrated care, said L. Scott Levin, MD, FACS, chair of the department of Orthopaedic Surgery. The goal is to provide patients with the same world-class care theyve come to expect at Penn Medicine, combined with added benefits and streamlined services to keep them engaged in their own care. When patients are involved and knowledgeable about their condition and the plan to management, both their experience and care are improved.

Developed using best practices from industry leaders in customer service, combined with Penn Medicine patient feedback, the Center offers a patient- and family-centered approach. For example, the Musculoskeletal Center provides each patient with a dedicated care coordinator, an innovative role that provides patients with a single point of contact for scheduling follow-up visits, additional tests, and referrals.

Upon arrival, patients visiting the Center will be escorted by a concierge to small waiting rooms which group patients according to specific conditions. For example, one hub is for patients with shoulder and elbow problems, while another is for knee injuries. In keeping with the goal of providing patients with a more engaging, personalized experience, the Center also offers added services such as check-in kiosks, iPads preloaded with injury-specific educational material, and large flat screens in exam rooms, to be used for clinicians to review and discuss imaging results with patients or offer patient education videos between visits with specialists.

Rehabilitation services are also a cornerstone of the facility, with Penn Therapy & Fitness University City providing specialized physical, occupational and speech therapy, including orthopedic and sports rehabilitation, hand therapy, cancer rehabilitation, neurologic therapy, cognitive therapy for patients recovering from strokes or head injuries, speech/language pathology, and womens health services. The facility contains Penn Medicines first therapy pool, complete with an underwater treadmill and underwater cameras. The cameras allow therapists to give guidance and direction while watching the patient move.

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Penn Medicine University City Unveiled: Market Street Tower is Primed to Boost Patient Engagement, Elevate the …

Google’s smart contact lenses for diabetics: Another step towards the Google-powered cyborg

Google X, the secretive research lab famed for its work on Glass and Googles self-driving cars, has unveiled its next product: Smart contact lenses. The first smart contact lens, aimed at diabetics, takes a glucose level reading every second from the tears that wash over your eye when you blink, and transmits that data wirelessly to a nearby device. Google doesnt say what device, but presumably a smartphone or Google Glass. Yes, if Google X has its way, we will soon all look like cyborgs, our bodies becoming interconnected networks of implants and augmentations.

Currently, if you have diabetes, you generally have to perform finger-stick blood tests throughout the day. At best this is inconvenient, at worst its disruptive and painful but either way, if you want to safely manage your blood sugar levels, it must be done. Due to the inconvenience factor, though, many diabetics dont test themselves as often as they should which then leads to all sorts of nasty situations, such as passing out. There are other ways of reliably testing blood sugar levels, such as your tears but, as you can imagine, collecting tears is no easier than pricking your finger. Unless you have a smart contact lens

Concept art for a multi-pixel wireless contact lens display.

With Googles smart contact lens equipped, if your glucose level dips or spikes, a warning can be transmitted instantly to a nearby display your smartphone, smartwatch, or head-mounted display (Google Glass).Future versions of the smart lens might even have a built-in LED that lights up if your glucose level crosses above or below a given threshold. This is obviously much easier and convenient than regularly sticking your finger. (Read:What is transhumanism, or, what does it mean to be human?)

Single pixel contact lens display, in a rabbits eye.

While it might sound incredibly advanced, the technology in Googles smart contact lens isnt that wild. Theres basically just two components, sandwiched between two layers of contact lens material: An antenna and rectifier, and a small glucose sensor. The problem with smart contact lenses, though, is the power source. Google glosses over this point because the current solution is rather ungraceful: Wireless power transfer from a nearby power source. Because of the tiny antenna, the power source probably has to be very close. Google Glass would work though, at this point in its development, were probably not talking about anything nearly as graceful its probably just stationary lab test. (Diabetics strapped to a chair, contact lens inserted, and then tested.)

The good news is, Google X has the best person in the world when it comes to smart contact lenses Babak Parviz. Way back in 2011, Parviz made a wireless smart contact lens with a single-pixel LED display(pictured right)Google X was obviously impressed with his work and picked him up. Smart contact lenses, due to rather unique placement theyre able to see the world, and display information to the wearer are one of the most exciting areas of cyborg implantation and augmentation. Their one weakness is that they are heavily constrained by their size and transparency requirement. Still, if Google X can find a graceful way of powering smart contact lenses, this could be a very exciting endeavor indeed.

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Google’s smart contact lenses for diabetics: Another step towards the Google-powered cyborg

ACGT® – Alliance for Cancer Gene Therapy – National Grants …

Better Quality of Life

In His Own Words: Another Patient Lives Because of Gene Therapy

Watch an Amazing Short Film of a Young Girl’s Recovery

Our donors have allowed top scientific minds to explore this new and promising avenue of cancer treatment, and their philanthropy is directly linked to the lives saved so far, Barbara Netter, ACGT president, said. Theres a lot more hope than there ever was. Its a very exciting time the beginning of the Golden Age, Netter described real progress being made through the results of research that have patients who faced dire diagnoses instead being in complete remissionnot short-term but for years and counting.

Read the full article at Connecticut Magazine

ACGT Scientific Advisory Council Chair Dr. Savio Woo introduced each of three ACGT Research Fellows who described how they are Achieving Cancer Remission with Cell and Gene Therapies. Dr. Carl June, University of Pennsylvania, Dr. Laurence Cooper, MD Anderson Cancer Center, and Dr. Michel Sadelain of Memorial Sloan-Kettering Cancer Center thanked ACGT for providing the initial funding that has enabled them to bring new therapies to patients. Read more…

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ACGT® – Alliance for Cancer Gene Therapy – National Grants …

ACGT Provides Seed Money for T-Cell Immunotherapy Cell and Gene Therapy Research; Successful Clinical Trials for …

ACGT today announced that seed money provided for innovative T-Cell immunotherapy cell and gene therapy research has led to two successful Clinical Trials for treatment of Acute Lymphoblastic Leukemia resulting in remissions in both children and adults who had no other hope of recovery.

Stamford, Connecticut (PRWEB) April 08, 2013

According to Michel Sadelain, MD, PhD, ACGT Scientific Advisory Council member and Research Fellow, and senior author of the Acute Lymphoblastic Leukemia study published March 21st in the journal Science Translational Medicine, This is a very exciting finding for patients and a major achievement in the field of targeted immunotherapy. Sadelain explained that these successful treatments would not have been possible had not been for the startup funding from ACGT. It wasnt easy to get funding to support such innovative, unproved therapies. The grant from ACGT was our first major grant to launch our T-cell program. Dr. Sadelain is the Director of the Center for Cell Engineering and Gene Transfer Expression Laboratory at Memorial Sloan-Kettering Cancer Center.

Dr. Sadelain, and Dr. Carl H. June, Perelman School of Medicine at the University of Pennsylvania, were each awarded $1 million in ACGT Clinical Investigator grants in 2004 to fund research using immune-mediated gene therapy for the treatment of lymphoma or leukemia. They were the first two Clinical Translational grants that ACGT awarded. Just last week, ACGT received 49 letters of intent, with a majority focused on T-cell therapy research, in response to its Request for Applications for 2013 Clinical Translational grants that will be awarded this Spring.

Dr. June, ACGT Scientific Advisory Council member and leader of the team that successfully treated two young leukemia patients, Emma Whitehead and Maddie Major, and seven other adult leukemia patients at the Abramson Cancer Center of the University of Pennsylvania (Penn) also commented on the cutting-edge research ACGT supported: “Funding was not available from the National Institutes of Health (NIH) for gene therapy (in 2004). ACGT was the first to be there, to say that cell and gene therapies can be a new platform therapy, and giving literally lifesaving grants to scientists who cannot get them from the federal system.”

ACGT is the only not-for-profit in the U.S. solely dedicated to cancer cell and gene therapy treatments for all types of cancer. 100% of contributions go directly to research. ACGT has funded 41 grants since its founding in 2001 totaling almost $24 million to fund both basic research and clinical translation. ACGT’s Scientific Advisory Council, comprised of 16 renowned physicians and researchers, conducts a rigorous review process. Seventeen ACGT funded research projects have been approved for human clinical trials, 11 of which are underway. To donate, please visit http://www.acgtfoundation.org or call 203.358.8000.

# # #

Press Contact:

Deborah Burns, 203.257.3163

deborah(at)burnscommunications(dot)net

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ACGT Provides Seed Money for T-Cell Immunotherapy Cell and Gene Therapy Research; Successful Clinical Trials for …

Northwestern Medicine researchers investigate stem cell therapy for stroke

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: Megan McCann memccann@nmh.org 312-926-5900 Northwestern Memorial Hospital

Each year, nearly 800,000 Americans suffer a stroke which can result in permanent brain damage, long term disability or death. As a leading cause of adult disability, stroke has an annual burden of more than $62 billion on the United States economy. With the exception of rehabilitation therapy, very few treatments are available to improve the chronic neurologic deficits caused by a stroke. In hopes of expanding therapeutic options, Northwestern Medicine researchers are investigating a novel stem cell therapy, known as SB623, that may hold the key to improving motor function following a disabling stroke.

Northwestern is currently one of only three sites in the nation enrolling participants in a landmark study to test the safety and efficacy of adult stem cell therapy for patients with stable ischemic stroke. Accounting for 87 percent of strokes, ischemic stroke occurs when a blocked artery interrupts the flow of oxygen and blood to the brain. This causes cell death and brain damage which can leave a person with impaired body functions, including paralysis, weakness on one side, difficulty with speech and language, vision issues, and cognitive challenges.

“Two million brain cells die each minute during a stroke making it critical to get treatment fast at the earliest sign of symptoms; once brain damage occurs, there’s very little that can be done medically to reverse it,” said principal investigator Joshua Rosenow, MD, director of Functional Neurosurgery at Northwestern Memorial Hospital and associate professor of neurosurgery, neurology, and physical medicine and rehabilitation at Northwestern University Feinberg School of Medicine. “While this study is only a preliminary step towards understanding the healing potential of these cells, we are excited about what a successful trial could do for a patient population that currently has very limited therapeutic options.”

While the study’s primary purpose is to examine the safety of SB623 stem cells, researchers will also seek to determine if the cells are effective in improving stroke symptoms. SB623 is derived from genetically engineered adult bone marrow cells from a healthy adult donor.

“Although not proven in humans, these stem cells have been shown to promote healing and improve function when administered in animal models of stable stroke,” said co-investigator Richard Bernstein, MD, director of Northwestern Memorial’s Stroke Center and associate professor of neurology at the Feinberg School. “The cells did not replace the neurons destroyed by stroke, but instead they appeared to encourage the brain to heal itself and promote the body’s natural regenerative process. Eventually, the implanted stem cells disappeared.”

“In this study, the cells are transplanted into the brain using brain mapping technology and scans, allowing us to precisely deposit the cells in the brain adjacent to the area damaged by stroke,” explained Rosenow.

Early participants have received 2.5 million cells, but as the study progresses the dose will escalate to 5 million and eventually 10 million cells. Since SB623 cells are allogeneic, a single donor’s cells can be used to treat many other individuals. Participants in the study will be followed for up to two years with periodic evaluations for safety and effectiveness in the improvement in motor function.

“Stroke can be a very disabling and life changing event,” said Bernstein. “Even just a slight improvement in function could make a huge difference for a person impacted by stroke. To potentially give our patients the opportunity to permanently regain movement or speech is a very exciting prospect. In the animal models, the improvements appeared to remain even after the implanted stem cells disappeared.”

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Northwestern Medicine researchers investigate stem cell therapy for stroke

Bombardier Aerospace Begins Production Of Its First Components In Morocco

Mon, Feb 11, 2013

Production is underway at the new Bombardier Aerospace transitional facility in Morocco. Following a graduation ceremony held on February 1 at the Institut des Metiers de l’Aeronautique (IMA), 18 aircraft assemblers began production of the first Bombardier components to come out of its Moroccan manufacturing facility.

“This is a very exciting milestone for us as we start to see our operation in Morocco take form,” said Hugo Brouillard, General Manager, Bombardier Aerospace, Morocco. “With 18 new local employees fully engaged and trained in the Bombardier manufacturing process and philosophy, this is the first step towards a long future of quality component manufacturing at another world-class Bombardier facility.”

Following the initial announcement in November 2011 of Bombardier’s intention to build a manufacturing facility in Morocco, the Company has extended its timeline for construction of the new facility as the manufacturing plan evolves. In order to maintain the timeline for production Bombardier has set up operations in a transitional facility located at the Mohammed V International Airport in Nouaceur in the Greater Casablanca region, near its future permanent facility.

The Bombardier Aerospace transitional manufacturing facility in Morocco is currently producing simple structures including flight controls for the CRJ Series aircraft. By year-end, the facility is expected to employ approximately 100 fully trained aircraft assemblers.

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Bombardier Aerospace Begins Production Of Its First Components In Morocco

Bombardier Aerospace Begins Production of the First Components in Morocco

MONTREAL, QUEBEC–(Marketwire – Feb 7, 2013) – Bombardier Aerospace today announced the start of production at its transitional facility in Morocco. Following a graduation ceremony held on February 1 at the Institut des Mtiers de l”Aronautique (IMA), 18 aircraft assemblers began production of the first Bombardier components to come out of its Moroccan manufacturing facility.

“This is a very exciting milestone for us as we start to see our operation in Morocco take form,” said Hugo Brouillard, General Manager, Bombardier Aerospace, Morocco. “With 18 new local employees fully engaged and trained in the Bombardier manufacturing process and philosophy, this is the first step towards a long future of quality component manufacturing at another world-class Bombardier facility.”

Following the initial announcement in November 2011 of Bombardier”s intention to build a manufacturing facility in Morocco, the Company has extended its timeline for construction of the new facility as the manufacturing plan evolves. In order to maintain the timeline for production Bombardier has set up operations in a transitional facility located at the Mohammed V International Airport in Nouaceur in the Greater Casablanca region, near its future permanent facility.

The Bombardier Aerospace transitional manufacturing facility in Morocco is currently producing simple structures including flight controls for the CRJ Series aircraft. By year-end, the facility is expected to employ approximately 100 fully trained aircraft assemblers.

About Bombardier

Bombardier is the world”s only manufacturer of both planes and trains. Looking far ahead while delivering today, Bombardier is evolving mobility worldwide by answering the call for more efficient, sustainable and enjoyable transportation everywhere. Our vehicles, services and, most of all, our employees are what make us a global leader in transportation.

Bombardier is headquartered in Montral, Canada. Our shares are traded on the Toronto Stock Exchange (BBD) and we are listed on the Dow Jones Sustainability World and North America indexes. In the fiscal year ended December 31, 2011, we posted revenues of $18.3 billion USD. News and information are available at bombardier.com or follow us on Twitter @Bombardier.

Note to editors

Follow @Bombardier_Aero on Twitter to receive the latest news and updates from Bombardier Aerospace.

Bombardier, CRJ and The Evolution of Mobility are trademarks of Bombardier Inc. or its subsidiaries.

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Bombardier Aerospace Begins Production of the First Components in Morocco

SpaceX Tests Precision Landing Rocket

Its been a busy week at SpaceX, with the company passing milestones for its manned space program, completing a test flight of its precision landing system and welcoming its latest Dragon back home from orbit.

The California company is finished with the first three performance milestones set out by NASA for the agencys Commercial Crew Integrated Capability program. The CCiCap initiative is similar to the commercial cargo development program SpaceX finished earlier this year, but instead the program is designed for carrying crew to low Earth orbit.

Right now the only way for NASA to send astronauts to the ISS is via the Russian Soyuz. From the beginning, SpaceX has designed both the Falcon 9 booster rocket and the Dragon spacecraft to be capable of manned space flight. And last week the company presented NASA with its plans for an integrated systems requirements review, the third milestone for the CCiCap program.

These initial milestones are just the beginning of a very exciting endeavor with SpaceX, said Ed Mango, NASAs Commercial Crew Program manager. We expect to see significant progress from our three CCiCap partners in a fairly short amount of time.

The two other companies working with NASA on the CCiCap are Boeing and Sierra Nevada Corporation.

Eventually SpaceX wants to employ a fully reusable space launch system. Currently, the Falcon 9 booster rocket isnt put back into service after splashing down in the Atlantic at the end of the first stage. The company has pointed out in the past that airlines dont throw away the airplane after every flight, and it believes one of most important achievements to greatly reducing the cost of orbital flight is to be able to reuse as much of the vehicle as possible after a launch.

In the future SpaceX plans to use rocket engines to allow the Falcon 9 to return to Earth in a controlled flight, with a precision landing at a specified landing site. On Saturday SpaceX founder Elon Musk sent out a video showing the latest (short) flight testing the companys precision landing rocket.

First flight of 10 story tall Grasshopper rocket using closed loop thrust vector & throttle control, Musk tweeted.

Similar to the previous Grasshopper flight, the rocket lifts off the pad for a short flight and returns safely to the ground. While the flight might not look like much, a controlled landing from even a low height as seen in the video is no easy task. The closed loop thrust vector and throttle are part of the automated controls for the Grasshopper. The Falcon 9 and Dragon both use automated controls for all phases of flight (with manual override capabilities) including the final docking with the ISS.

The Grasshopper is a suborbital test vehicle with a wider, landing gear structure at its base to allow for vertical take off and landing capability. Eventually the landing gear is expected to fold up flush with the first stage during flight and be extended again for landing.

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SpaceX Tests Precision Landing Rocket

BEST SELLER IN A WEEKEND SPOTLIGHT, FAIRMONT HOTEL, SAN FRA – Video



BEST SELLER IN A WEEKEND SPOTLIGHT, FAIRMONT HOTEL, SAN FRA
Ana Maria Sanchez – I was a Featured Author speaking on all the marvelous changes my life has undergone after becoming a bestselling author. THANKS TO ALL OF YOU I made best seller status last year on Amazon in two categories 1.Spirituality 2. Women's Wellness. It was a very exciting time and continues to be so. Thanks to my book publisher, business coach, marketing expert, mentor and all around friend Alicia Dunams. http://www.aliciadunams.comFrom:juliekphotoViews:1 0ratingsTime:02:09More inFilm Animation

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BEST SELLER IN A WEEKEND SPOTLIGHT, FAIRMONT HOTEL, SAN FRA – Video

Hard to believe what I went through to make a dream come true – Video



Hard to believe what I went through to make a dream come true
http://www.englishlanguagegames.org For the first time in my life, I'm actually really proud of myself for what I've accomplished and am doing. I absolutely LOVE teaching ESL beginners, my students range from 2 to 42 years old! And the best part is that because it's my own school I have the freedom to create my own English teaching materials, which has been a very exciting and creative adventure!From:englishlanguagegamesViews:46 0ratingsTime:04:13More inEducation

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Hard to believe what I went through to make a dream come true – Video

Private Dragon Spacecraft 'Go' to Launch Toward Space Station Sunday

A private Dragon space capsule is poised for a weekend launch to the International Space Station with the first big cargo shipment ever aboard an unmanned American spacecraft.

The gumdrop-shaped Dragon spacecraft, built by the commercial spaceflight company SpaceX, will blast off on Sunday (Oct. 7) from a launch pad at Cape Canaveral Air Force Station in Florida. Liftoff is set for 8:35 p.m. EDT (0035 Monday GMT), with a planned arrival at the station set for on Wednesday (Oct. 10).

SpaceX and NASA mission managers met Friday to review the preparations for the Dragon flight atop its Falcon 9 rocket, ultimately giving the mission a final “go” for launch. There is a 60 percent chance of good weather for launch.

“It’s going to be a very exciting night on Sunday,” SpaceX founder Elon Musk said during a Google+ hangout event with NASA chief Charles Bolden today ahead of the review. “I always get kind of nervous before these flights, thinking, like ‘What have we missed?'” [SpaceX’s Dragon Poised to Sunday Launch (Photos)]

Musk said he and his SpaceX team have done their best to ensure a successful launch Sunday, and while there is always the chance of something going wrong, “I feel like we’ve done everything we can to make the mission as successful as possible, and I hope people enjoy watching it.”

Private delivery for space station

The Dragon mission, the first official cargo run by a private American-built spacecraft, is a watershed flight for NASA and SpaceX. The Hawthorne, Calif.-based company has a $1.6 billion deal with NASA for at least 12 resupply missions to the space station using its robotic Dragon capsules and their Falcon 9 rocket boosters.

The deal is part of NASA’s Commercial Resupply Services (CRS) program. With the retirement of NASA’s space shuttle fleet last year, the space agency is depending on the availability of new private space taxis to provide unmanned cargo deliveries to the station, as well as ferry American astronauts to and from low-Earth orbit.

Another U.S. company, the Virginia-based Orbital Sciences Corp., has a $1.9 billion agreement to fly at least eight resupply missions to the station using its own Antares rockets and Cygnus spacecraft, and plans to launch a rocket test flight later this year. But SpaceX is the first of the two firms to actually launch vehicles to the International Space Station.

In May, SpaceX (short for Space Exploration Technologies) launched a different Dragon capsule and Falcon 9 rocket on a test flight to the station. That mission demonstrated SpaceX’s ability to launch Dragon to the station, have it rendezvous with the orbiting lab safely, and then be captured for docking using a robotic arm controlled by astronauts inside the outpost. [SpaceX’s 1st Dragon Flight to Space Station (Video)]

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Private Dragon Spacecraft 'Go' to Launch Toward Space Station Sunday

Soyuz docks with space station, boosts crew back to six

A Russian Soyuz spacecraft carrying a cosmonaut, a NASA astronaut, and a Japanese engineer — all space veterans — docked with the International Space Station early Tuesday after a flawless rendezvous.

A Russian Soyuz ferry craft glided to a computer-orchestrated docking with the International Space Station early Tuesday, bringing a Russian cosmonaut, a NASA astronaut and a Japanese engineer to the lab complex two days after launch from the Baikonur Cosmodrome in Kazakhstan.

The Soyuz TMA-05M spacecraft as seen from the space station early Tuesday during the final phases of a two-day rendezvous.

With veteran cosmonaut Yuri Malenchenko monitoring the autonomous approach from the center seat of the Soyuz TMA-05M command module, the spacecraft’s docking system engaged its counterpart on the Earth-facing Rassvet module at 12:51 a.m. EDT as the two spacecraft sailed 252 miles above northeast Kazakhstan.

“Docking confirmed,” someone said on the Russian flight control loop.

A few moments later, the docking mechanism pulled the Soyuz snugly into place for extensive leak checks, standard procedure before hatches could be opened.

The view of the International Space Station from an approaching Soyuz spacecraft bringing three fresh crew members to the lab complex.

The linkup occurred on the 37th anniversary of the Apollo-Soyuz Test Program docking in 1975, the first joint U.S.-Russian space mission.

“Congratulations to our crew and to our Russian partners,” said Mike Surber, director of NASA operations in Russia. “It’s a very exciting day as it’s the 37th anniversary of the handshake between our two countries during Apollo-Soyuz. And here’s (hoping) for 37 more years.”

Malenchenko, NASA flight engineer Sunita Williams and Japanese astronaut Akihiko Hoshide were joining Expedition 32 commander Gennady Padalka, cosmonaut Sergei Revin and NASA astronaut Joseph Acaba, who were launched to the space station May 15. They’ve had the outpost to themselves since July 1 when three earlier crew members departed and returned to Earth.

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Soyuz docks with space station, boosts crew back to six

NASA Space Launch System Core Stage Moves From Concept to Design

HUNTSVILLE, Ala. — The nation’s space exploration program is taking a critical step forward with a successful major technical review of the core stage of the Space Launch System (SLS), the rocket that will take astronauts farther into space than ever before.

The core stage is the heart of the heavy-lift launch vehicle. It will stand more than 200 feet (61 meters) tall with a diameter of 27.5 feet (8.4 meters).

NASA’s Marshall Space Flight Center in Huntsville, Ala., hosted a comprehensive review. Engineers from NASA and The Boeing Co. of Huntsville presented a full set of system requirements, design concepts and production approaches to technical reviewers and the independent review board.

“This meeting validates our design requirements for the core stage of the nation’s heavy-lift rocket and is the first major checkpoint for our team,” said Tony Lavoie, manager of the SLS Stages Element at Marshall. “Getting to this point took a lot of hard work, and I’m proud of the collaboration between NASA and our partners at Boeing. Now that we have completed this review, we go from requirements to real blueprints. We are right on track to deliver the core stage for the SLS program.”

The core stage will store liquid hydrogen and liquid oxygen to feed the rocket’s four RS-25 engines, all of which will be former space shuttle main engines for the first few flights. The SLS Program has an inventory of 16 RS-25 flight engines that successfully operated for the life of the Space Shuttle Program. Like the space shuttle, SLS also will be powered initially by two solid rocket boosters on the sides of the launch vehicle.

The SLS will launch NASA’s Orion spacecraft and other payloads, and provide an entirely new capability for human exploration beyond low Earth orbit. Designed to be safe, affordable and flexible for crew and cargo missions, the SLS will continue America’s journey of discovery and exploration to destinations including nearby asteroids, Lagrange points, the moon and ultimately, Mars.

“This is a very exciting time for the country and NASA as important achievements are made on the most advanced hardware ever designed for human spaceflight,” said William Gerstenmaier, associate administrator for the Human Exploration Operations Mission Directorate at NASA Headquarters in Washington. “The SLS will power a new generation of exploration missions beyond low Earth orbit and the moon, pushing the frontiers of discovery forward. The innovations being made now, and the hardware being delivered and tested, are all testaments to the ability of the U.S. aerospace workforce to make the dream of deeper solar system exploration by humans a reality in our lifetimes.”

The first test flight of NASA’s Space Launch System, which will feature a configuration for a 77-ton (70-metric-ton) lift capacity, is scheduled for 2017. As SLS evolves, a two-stage launch vehicle configuration will provide a lift capability of 143 tons (130 metric tons) to enable missions beyond low Earth orbit and support deep space exploration.

Boeing is the prime contractor for the SLS core stage, including its avionics. The core stage will be built at NASA’s Michoud Assembly Facility in New Orleans using state-of-the-art manufacturing equipment. Marshall manages the SLS Program for the agency.

Across the SLS Program, swift progress is being made on several elements. The J-2X upper-stage rocket engine, developed by Pratt & Whitney Rocketdyne for the future two-stage SLS, is being tested at Stennis Space Center in Mississippi. The prime contractor for the five-segment solid rocket boosters, ATK of Brigham City, Utah, has begun processing its first SLS hardware components in preparation for an initial qualification test in 2013.

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NASA Space Launch System Core Stage Moves From Concept to Design

F.X. Matt Brewery installs 30,000 pound stainless steel digester tank

A 30,000 pound stainless steel digester tank was lifted in the air and installed as part of the F.X. Matt Brewery’s new anaerobic digester system Wednesday morning. The eco-friendly system will break down waste materials into methane and will be used to generate electricity and heat for the facility. The facility should be completed by the fall, officials said. “This is a very exciting project,” said Nick Matt, Chairman and CEO of the F.X. Matt Brewery. The company will use the technology to clean the brewery’s wastewater by about 80 to 90 percent. Electricity generated from the process will account for about 30 to 40 percent of the company’s usage. Local officials like Mayor Robert Palmieri and Oneida County Executive Anthony Picente attended the event. “The Matts have been absolutely legendary in the City of Utica for decades,” Palmieri said. “They’re investing and they’re loyal and passionate about this community.”

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F.X. Matt Brewery installs 30,000 pound stainless steel digester tank