Pinoy scientist studies genes to treat bad eyes

SCIENTISTS in Florida, USA, are studying gene therapy to cure genetic eye diseases.

Prof. Galileo Encabo, senior biological scientist at the Department of Opthalmology of the University of Florida, said they have conducted successful human clinical trials, but it would take years before the technology can be made available to the public.

From clinical trials, it takes a few years to make it available for public use. We will have to find a pharmaceutical firm willing to produce (the technology), then we have to get approval from the different bureaucrats, Encabo told reporters.

Gene therapy is the introduction of new genes into human cells to treat a disease. It involves replacing, correcting or removing an abnormal gene.

Encabo held a seminar at the University of the Philippines Cebu yesterday. He was a graduate and a former professor of the university.

The two-hour seminar, held at the UP Cebu Union building, was organized by the UP Cebu Central Visayas Studies Center in partnership with the universitys Sciences Cluster.

It aims to inspire students to pursue studies on molecular genetics.

The University of Florida is collaborating with 65 other institutions in its research on gene therapy.

Encabo said the persons who were subjected to the clinical trials improved their vision, with some increasing their visual ability 10,000 times.

The success of the human clinical trials, he said, will attract more private organizations to support the experiments and validate the whole concept of human gene therapy.

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Pinoy scientist studies genes to treat bad eyes

The State of Gene Therapy in a Biogen Idec World: Sangamo, Bluebird and Acceleron

As part of my 2014 predictions, I argued gene therapy would get hot and investor interest would turn toward the monogenic programs at Sangamo Biosciences (SGMO). The partnership announced on Jan. 9 between Biogen Idec (BIIB) and Sangamo confirmed my prediction to a certain extent, albeit sooner in the year than I expected.

Let's discuss what the Biogen partnership means for Sangamo moving forward, and discuss the implications for other gene therapy stocks like Bluebird (BLUE) and Acceleron (XLRN)?

The partnership covers two Sangamo programs: sickle cell disease (SCD) and beta-thalassemia (BT). Sangamo will receive a $20 million upfront payment and is eligible for just about $300 million in future milestones. While it is not explicitly stated, $15 million of those milestones appear to be related to the start of a phase I trial. Sangamo will receive double-digit royalties on sales and retains the right to co-promote in the U.S., assuming drug approval, of course.

Summed up, the new partnership is a nice entry into the gene therapy business for Biogen and continued validation of the Sangamo monogenic disease pipeline.

Sobek has no position in any stocks mentioned in this column.

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The State of Gene Therapy in a Biogen Idec World: Sangamo, Bluebird and Acceleron

Gene therapy leads to robust improvements in animal model of fatal muscle disease

PUBLIC RELEASE DATE:

22-Jan-2014

Contact: Kim Blakely krb13@uw.edu 206-685-1323 University of Washington

Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease.

Researchers, based at the University of Washington, Seattle, Washington, Gnthon, France, Boston Children's Hospital in Massachusetts, and Virginia Polytechnic Institute and State University (Virginia Tech) in Blacksburg, Virginia, conducted the study. The scientists found that both mice and dogs responded from a single intravascular injection of AAV, produced at Gnthon, with robust improvement in muscle strength, corrected muscle structure at the microscopic level, and prolonged life. No toxic or immune response was observed in the dogs.

These results demonstrate the efficacy of gene replacement therapy for myotubular myopathy in animal models and pave the way to a clinical trial in patients.

Children born with X-linked myotubular myopathy, which affects about 1 in 50,000 male births, have very weak skeletal muscles, causing them to appear floppy, with severe respiratory difficulties. Survival beyond birth requires intensive support, often including tube feeding and mechanical ventilation, but effective therapy is not available for patients, and most die in childhood.

Alan H. Beggs of Boston Children's Hospital, co-senior author on the paper, has studied the mutated gene, known as MTM1, for many years and previously showed that replacing missing myotubularin protein effectively improved MTM muscles' ability to contract.

Based on seminal work on local and systemic administration in a mouse model of the disease performed by Anna Buj-Bello at Gnthon since 2009, Martin K. Childers, a professor of rehabilitation medicine and a regenerative medicine researcher at the University of Washington, worked with the Buj-Bello and Beggs groups.

They tested gene therapy using an engineered adenovirus vector, created by Gnthon. The vector is a vehicle for delivering a replacement MTM1 gene into cells. The researchers used two animal models: mice with an engineered MTM1 mutation and dogs carrying a naturally occurring MTM1 gene mutation. These mutant animals appear very weak with shortened lifespans, similar to patients with myotubular myopathy.

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Gene therapy leads to robust improvements in animal model of fatal muscle disease

Thinking Strategically About Intelligent Decision Management: Technology Futurist/Speaker Jack Shaw – Video


Thinking Strategically About Intelligent Decision Management: Technology Futurist/Speaker Jack Shaw
Hi. I #39;m Jack Shaw, the Business Technology Futurist. This is one in a series of brief videos in which I will discuss intelligent systems and how you, and you...

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Thinking Strategically About Intelligent Decision Management: Technology Futurist/Speaker Jack Shaw - Video

The Paleofuture Blog Is 7 Years Old Today

S

The Paleofuture blog turns 7 years old today! And you can now find every Paleofuture post ever writtenright here at Gizmodo.

When I came on board at Gizmodo this past May, I brought my entire Paleofuture archive with me. Every post I've ever written under the Paleofuture nameat Paleofuture.com and Smithsoniancan now be found here at Paleofuture dot Gizmodo dot com. All you need to do is use that little search bar in the upper lefthand corner.

For instance, if you're interested in Nikola Tesla's predictions for the future, just type in "Tesla." Interested in robots or monorails or Disney? Same deal.

Paleofuture explores how people of the past imagined the future, and we're always making more futurism. So conceivably I could do this forever. But we'll see about that.

We're seven years in and (amazingly) I'm not sick of the topic yet. I really do think that futurism provides the most fascinating lens through which to study historyfrom the shiny plastic utopias of the 1950s to the dark polluted dystopias of the 1970s.

And as I've said before, looking at the accuracy of past predictions is really just a jumping off point. Determining if an old prediction was "right" is fun, but the truly interesting question is why people made their particular prediction to begin with. What was the context of the prediction? What were people excited or terrified about? As always, predictions say more about the person making them than they do about the actual future.

So here's to seven more years of our weird and wonderful visions of yestermorrow, and definitely drop me a line (novak@gizmodo.com) if you find something paleofuture-y that I haven't covered.

Thanks for reading.

Image: Scanned from the April 27, 1958 edition of Arthur Radebaugh's Sunday comic strip "Closer Than We Think"

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The Paleofuture Blog Is 7 Years Old Today

Assassin’s Creed IV Freedom Cry Detonado Parte 8: Investigação Científica [1080p PT-BR] – Video


Assassin #39;s Creed IV Freedom Cry Detonado Parte 8: Investigao Cientfica [1080p PT-BR]
Assassin #39;s Creed IV Black Flag Freedom Cry DLC - Parte 8 Investigao Cientfica 100% Sincronizao (Grito de Liberdade DLC) Gameplay 1080p HD PC PS4 Xbox On...

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Assassin's Creed IV Freedom Cry Detonado Parte 8: Investigação Científica [1080p PT-BR] - Video

Assassin’s Creed IV Freedom Cry Detonado Parte 9: Explorando o Mar [1080p PT-BR] – Video


Assassin #39;s Creed IV Freedom Cry Detonado Parte 9: Explorando o Mar [1080p PT-BR]
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Assassin's Creed IV Freedom Cry Detonado Parte 9: Explorando o Mar [1080p PT-BR] - Video

Assassin’s Creed IV Freedom Cry Detonado Parte 11: Plante as Sementes [1080p PT-BR] – Video


Assassin #39;s Creed IV Freedom Cry Detonado Parte 11: Plante as Sementes [1080p PT-BR]
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Assassin’s Creed IV Freedom Cry Detonado Parte 10: Wellington e Montez [1080p PT-BR] – Video


Assassin #39;s Creed IV Freedom Cry Detonado Parte 10: Wellington e Montez [1080p PT-BR]
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Assassin's Creed IV Freedom Cry Detonado Parte 10: Wellington e Montez [1080p PT-BR] - Video

Report: Freedom Declined Worldwide in 2013

WASHINGTON The Washington-based group Freedom House said the cause of freedom around the globe took another hit in 2013, as the group released its Freedom in the World 2014 report Thursday. The authors warned of serious setbacks in some of the worlds more influential countries. Perhaps nowhere was the fall from freedom more visible than in Egypt, where angry protests gave way to a military coup and a crackdown on dissent. Freedom in the World 2014 report author Arch Puddington argued that in many ways, Egypt was not alone. We are at a time right now where the leaders of the authoritarian community are more self-assured and arrogant than theyve been in the past and theres a kind of a loose coalition, alliance of the repressive countries, said Puddington. Color revolutions

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Almost no region fared worse than Eurasia, which saw increased persecution and media crackdowns in Russia and Ukraine. According to Freedom House, 78 percent of the regions population lives in countries that are "not free.

x

Freedom House said democracies, especially the U.S., also seem plagued by self-doubt, unable or unwilling to act, a condition creating adverse outcomes elsewhere in the world. The group cited the lack of progress in the Middle East as a prime example. The Washington Institutes David Schenker said surveys suggest U.S. citizens are more interested in internal affairs. The United States public is not at all interested in having a more, military involvement in the Middle East or even financial involvement or diplomatic involvement.Weve seen that in the polling.Theyre more focused on domestic matters, said Schenker. You still have advocates and activists and human rights defenders in many places around the world looking to the U.S. for leadership and theyre just not finding it the way they would like.

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Report: Freedom Declined Worldwide in 2013

Taiwan celebrates the 2014 World Freedom Day

TAIPEI, Taiwan - Today is World Freedom Day, which marks the kick off for several celebrations in the week to commemorate this special day.

World Freedom Day is especially celebrated in Taiwan and South Korea due to the fact that Jan. 23, 1954 was the return date for prisoners in the Korean War.

As those soldiers landed in Keelung, they received warm greetings from Taiwanese people.

People also rang the freedom bell 23 times in every city in Taiwan in celebration. The Republic of China established the date as World Freedom Day ever since then.

As it was the 60th anniversary of the return of these soldiers, the Taipei Veterans Home held a celebration yesterday and invited the Taipei Jingmei Girls High School honour guard and colour guard to perform for retired soldiers.

Hsiung Chi-chih, manager of the Taipei Veterans Home, said a lot of people had already forgotten this history. The decision to come to Taiwan that these soldiers made actually played an important role in stabilizing the government at that time.

Therefore, it is an event worthy of remembrance, stated Hsiung.

In order to help more people understand this history, Hsiung also decided to publish a commemorative album. The main goal of celebration activities is to let people reflect on our past and think about the true essence of democracy and freedom.

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Taiwan celebrates the 2014 World Freedom Day

UPDATE: Freedom Industries Responds to WVDEP, Two Chemicals Leaked from Tank

Freedom Response to WVDEP Order

WVDEP Order for Freedom Industries

UPDATE 1/22/14 @ 5:10 p.m. CHARLESTON, W.Va. (WSAZ) -- Freedom Industries says chemicals MCHM and PPH were the only two chemicals that leaked from a tank on their property into the Elk River earlier this month.

The leak, that happened on January 9, led to a state of emergency and "do not use" water ban for West Virginia American Water customers in nine West Virginia counties.

It wasn't until January 21 that the company told the DEP that PPH also leaked from the tank.

PPH is added to the Crude MCHM to act as an extender in that the Crude MCHM is available in limited, sporadic quantities, Freedom said in its response letter to the WVDEP. At the time of the release on Jan. 9, the blend in Tank No. 396, after extensive calculation, was approximately 88.5 percent Crude MCHM, and 7.3 percent PPH by weight and 4.2 percent water by weight. Our records and internal investigations indicate that there were no other materials in Tank 396 at the time of the release.

Freedoms letter was signed by Freedom President Gary Southern.

Freedom told the WVDEP on Jan. 21 that the storage tank contained roughly 300 gallons of PPH that it adds to its Crude MCHM mixture.

This material was not included in the initial information regarding the composition of the spilled materials. Failure to accurately report the type or types and quantity or quantities of the material or materials therein is a violation of state code, according to the DEP.

Having this revelation so late in the game is completely unacceptable, said WVDEP Cabinet Secretary Randy Huffman said earlier today. We have ordered Freedom to reveal any other information they have regarding the contents of the tank that leaked.

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UPDATE: Freedom Industries Responds to WVDEP, Two Chemicals Leaked from Tank