Archive for the ‘Gene Therapy Research’ Category

PUBLIC RELEASE DATE:

27-Jan-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, January 27, 2014Drugs comprised of single strands of DNA, called aptamers, can bind to targets inside tumor cells causing cell death. But these DNA drugs cannot readily get inside tumor cells on their own. Effective delivery of DNA aptamers using a natural polysaccharide as a carrier is described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. The article is available on the Nucleic Acid Therapeutics website.

Tatyana Zamay and coauthors, Krasnoyarsk State Medical University, Siberian Branch Russian Academy of Sciences, and Center for Reproductive Medicine (Krasnoyarsk, Russia), and University of Ottawa, Canada, combined the polysaccharide arabinogalactan, obtained from the larch tree, with a DNA drug that binds to and disrupts the activity of vimentin, a structural protein required for cell division. Vimentin is often over-produced by tumor cells compared to normal cells.

In the article "DNA-Aptamer Targeting Vimentin for Tumor Therapy in Vivo" the authors show that an aptamer targeting vimentin inhibits tumor growth more effectively when it is administered as a mixture with arabinogalactan than alone.

"This work demonstrates the advancement of aptamer therapeutic application through increased bioavailability using a nontoxic polysaccharide based therapy," says Executive Editor Graham C. Parker, PhD.

###

Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Graham C. Parker, PhD.

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A natural sugar delivers DNA aptamer drug inside tumor cells

By REBECCA GOURLEY WNPA Olympia News Service

OLYMPIA Fish farms in Washington are still not happy with an updated bill that intends to label transgenic fish.

Transgenic is a form of genetic engineering where the DNA is spliced to create more desirable traits.Although the new bill only addresses labeling and not the production of these fish, the industry opposes it.

Rep. Cary Condotta, R-East Wenatchee, originally filed House Bill 2143, which would prohibit the production of genetically modified finfish and would require them to be labeled when sent to supermarket shelves to be purchased as food by consumers.

Condotta has since filed House Bill 2630, which has similar language but one big difference: It doesnt prohibit production of transgenic fish.

Condotta said that because the production of transgenic fish is already banned in Washington's marine waters, including a ban in the bill was unnecessary. But people in Washington support the labeling of transgenic fish, he said, so its an issue that legislators should address.

The simplified bill also will be better for the state's aquaculture industry, because they should be concerned about their products getting mistaken for transgenic products, he said.

After listening to fish farmers criticize his original bill at a Jan. 17 hearing before the House Agriculture and Natural Resources committee, Condotta had said he was surprised by their opposition.

"We thought that the farmed fishermen would be on our side," he said, considering that several aquaculture companies have said they have no plans to rear transgenic fish in the future.

However, support for the bill was not coming from Troutlodge, an aquaculture company based in Bonney Lake, southeast of Tacoma.

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New GMO-labeling bill fails to appease fish farms

OLYMPIA Fish farms in Washington are still not happy with an updated bill that intends to label transgenic fish.

Transgenic is a form of genetic engineering where the DNA is spliced to create more desirable traits. Although the new bill only addresses labeling and not the production of these fish, the industry opposes it.

Rep. Cary Condotta, R-East Wenatchee, originally filed House Bill 2143, which would prohibit the production of genetically modified finfish and would require them to be labeled when sent to supermarket shelves to be purchased as food by consumers.

Condotta has since filed House Bill 2630, which has similar language but one big difference: It doesnt prohibit production of transgenic fish.

Condotta said that because the production of transgenic fish is already banned in Washington's marine waters, including a ban in the bill was unnecessary.

But people in Washington support the labeling of transgenic fish, he said, so its an issue that legislators should address.

The simplified bill also will be better for the state's aquaculture industry, because they should be concerned about their products getting mistaken for transgenic products, he said.

After listening to fish farmers criticize his original bill at a Jan. 17 hearing before the House Agriculture and Natural Resources committee, Condotta had said he was surprised by their opposition.

"We thought that the farmed fishermen would be on our side," he said, considering that several aquaculture companies have said they have no plans to rear transgenic fish in the future.

However, support for the bill was not coming from Troutlodge, an aquaculture company based in Bonney Lake, southeast of Tacoma.

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Rep. Condotta responds to concerned fisheries with revamped GMO bill

Study linking belly fat to genetics could lead to cure for obesity
A study at the University of Louisville linking belly fat to genetics could lead to a cure for obesity.

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Study linking belly fat to genetics could lead to cure for obesity - Video

Savage Genetics - Massacre (Clip)
Clip of a tune i #39;ve been sat on for a wile i #39;m hoping to get a few tunes together for a free mini EP, as for when this will happen i #39;m not certain but keep a...

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Bio 8.2 Mendelian Genetics Introduction

By: Heather Newman

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Doctor Oz Show, would you guess me to be 41 years old? 60849
http://bit.ly/1i3Ahld Doctor Oz, can you tell how old I REALLY am? Can skin cell therapy make me feel AND LOOK younger again? Doctor Oz Show, would you guess...

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Abandoned dog receives historic stem cell therapy
Veterinarians across the country now have a way to improve the lives of their patients by using a tool to combat osteoarthritis. On Thursday, one dog made hi...

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Autologous Cell Therapy Market
For Details : http://goo.gl/56kgZS Autologous Cell Therapy Market reserach report gives a detailed analysis about state of the art of both autologous stem ce...

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ELLSWORTH Visitors to the Bellaire pet crisis center With a Little help From My Friends get an official welcome from Moka.

The Labrador retriever was found behind a Bellaire restaurant in 2011 and now serves as the centers mascot.

Peforming her duties has been increasingly difficult for the dog, who suffers from severe arthritis in her hips. So recently the center turned to Ellsworth veterinarian Christian Randall of North Country Veterinary Services, the first in northern Michigan to offer in-clinic adipose stem cell therapy.

The procedure uses a pets own blood and tissue to produce plasma-rich platelets and stem cells that proliferate growth in damaged areas.

Dormant stem cells are separated from adipose -- fat tissue -- and activated with an LED technology that uses three different wave lengths of light. Then the cells are injected directly into the affected area or administered intravenously to help promote regeneration. The result is a decrease in pain and lameness and increased range of motion.

Its using the bodys own repair cells to repair damage, said Trey Smith, director of laboratory services for MediVet America, which developed the technology Randall uses.

The therapy is the first treatment to help heal and slow the progression of osteoarthritis and degenerative joint disease rather than just cope with the symptoms, said Randall, who saw the results while studying at Virginia Equine Imaging and now plans to use it on equine as well as canine and feline patients.

It concentrates, speeds up and amplifies the bodys own healing power, he said.

Stem cell therapy has been around for a while, but in-clinic availability of the technology is new. Only a handful of veterinarians in Ann Arbor and Grand Rapids offer the services, said Randall, who charges $1,800 to treat a dog or cat. Repeat injections are possible with banked plasma-rich platelets and stem cells.

Before the one-day procedure, veterinarians had to send blood and tissue to an outside lab for processing, a more costly three-day procedure that requires an animal's return visit to the vet for injection.

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Stem-cell therapy restores faith for arthritic pets

PUBLIC RELEASE DATE:

26-Jan-2014

Contact: Sue McGreevey smcgreevey@partners.org 617-724-2764 Massachusetts General Hospital

A simple adjustment to a powerful gene-editing tool may be able to improve its specificity. In a report receiving advance online publication in Nature Biotechnology, Massachusetts General Hospital (MGH) investigators describe how adjusting the length of the the guide RNA (gRNA) component of the synthetic enzymes called CRISPR-Cas RNA-guided nucleases (RGNs) can substantially reduce the occurrence of DNA mutations at sites other than the intended target, a limitation the team had previously described just last year.

"Simply by shortening the length of the gRNA targeting region, we saw reductions in the frequencies of unwanted mutations at all of the previously known off-target sites we examined," says J. Keith Joung, MD, PhD, associate chief for Research in the MGH Department of Pathology and senior author of the report. "Some sites showed decreases in mutation frequency of 5,000-fold or more, compared with full length gRNAs, and importantly these truncated gRNAs - which we call tru-gRNAs - are just as efficient as full-length gRNAs at reaching their intended target DNA segments."

CRISPR-Cas RGNs combine a gene-cutting enzyme called Cas9 with a short RNA segment and are used to induce breaks in a complementary DNA segment in order to introduce genetic changes. Last year Joung's team reported finding that, in human cells, CRISPR-Cas RGNs could also cause mutations in DNA sequences with differences of up to five nucleotides from the target, which could seriously limit the proteins' clinical usefulness. The team followed up those findings by investigating a hypothesis that could seem counterintuitive, that shortening the gRNA segment might reduce off-target mutations.

"Some of our experiments from last year suggested that one could mismatch a few nucleotides at one end of the gRNA complementarity region without affecting the targeting activity," Joung explains. "That led us to wonder whether removing these nucleotides could make the system more sensitive to mismatches in the remaining sequence."

Based on a natural system a species of bacteria uses against other pathogens, the CRISPR-Cas RGNs most widely used by researchers includes a 20-nucleotide targeting region within the gRNA. To test their theory, the MGH team constructed RGNs with progressively shorter gRNAs and found that, while gRNAs with targeting segments of 17 or 18 nucleotides were as or more efficient than full-length gRNAs in reaching their targets, those with 15- or 16-nucleotide targeting segments had reduced or no targeting activity. Subsequent experiments found that 17-nucleotide truncated RGNs efficiently induced the desired mutations in human cells with greatly reduced or undetectable off-target effects, even at sites with only one or two mismatches.

"While we don't fully understand the mechanism by which tru-gRNAs reduce off-target effects, our hypothesis is that the original system might have more energy than it needs, enabling it to cleave even imperfectly matched sites," says Joung, who is an associate professor of Pathology at Harvard Medical School. "By shortening the gRNA, we may reduce the energy to a level just sufficient for on-target activity, making the nuclease less able to cleave off-target sites. But more work is needed to define exactly why tru-gRNAs have reduced off-target effects."

###

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Shortening guide RNA markedly improves specificity of CRISPR-Cas nucleases

Inject a gene from a certain cold-water fish into a strawberry, and the strawberry can withstand colder temperatures. But would you still want to eat it?

Such advances in genetic engineering have implications for helping feed a growing, hungry world but a lot of people aren't too keen on eating those advances just yet.

Others wouldn't hesitate.

The difference reflects the "wild, messy debate" surrounding genetically modified food, with one of the more recent skirmishes centering on whether food labels should contain information about such ingredients, according to Nick George, president of the Midwest Food Processors Association, based in Madison.

Wisconsin's agriculture and food production industries find themselves smack in the middle of the debate.

"This is a big issue," George said. "It's not going away."

Neither, it seems, are genetically engineered crops in the American food chain.

The U.S. Department of Agriculture estimates that 93% of soybean acres and 85% of corn acres in 2013 were planted with genetically modified, herbicide-tolerant crop varieties.

The percentage of insect-resistant corn planted in 2013 stood at 76%, according to the USDA. The insect-resistant corn contains a gene from the soil bacterium Bt Bacillus thuringiensis. The bacteria produce a protein that is toxic to specific insects.

Consider that there are nearly 1.3 million dairy cows in Wisconsin, and some of them are no doubt eating corn with genetically modified ingredients.

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Debate rages over labeling genetically modified food

Startup Global Bioenergies uses genetic engineering to avoid one of the costliest steps in biofuel production.

Test plant: Global Bioenergies is adapting this test facility, owned by the European research organization Fraunhofer, to produce biofuels using a new process that avoids a costly distillation step.

Audi is investing in a startup, Paris-based Global Bioenergies, that says it can make cheap gasoline from sugar and other renewable sources. The strategic partnership includes stock options and an unspecified amount of funding.

As with conventional biofuel production, Global Bioenergies technology uses microrganisms to ferment sugars to produce fuel. But its process eliminates the second most costly part of producing biofuelsthe energy-intensive distillation step. And by making gasoline instead of making ethanol, the startup skirts a major problem hampering growth in biofuelsthe fact that the market for ethanol is saturated.

Global Bioenergies has demonstrated its technology in the lab and is building two pilot facilities to produce isobutene, a hydrocarbon that a partner will convert into gasoline through an existing chemical process. The larger of the two pilot facilities will be big enough to support the production of over 100,000 liters of gasoline a year.

The process addresses one of the key challenges with conventional biofuels productionthe fuel can kill the microrganisms that make it. In a conventional fermentation process, once the concentration of ethanol gets to about 12 percent, it starts to poison the yeast so that it cant make any more ethanol.

Global Bioenergies has genetically engineered E. coli bacteria to produce a gas (isobutene) that bubbles out of solution, so its concentration in the fermentation tank never reaches toxic levels. As a result the bacteria can go on producing fuel longer than in the conventional process, increasing the output of a plant and reducing capital costs.

The isobutene still needs to be separated from other gases such as carbon dioxide, but Global Energies says this is much cheaper than distillation.

The new process doesnt address the biggest cost of biofuels todaythe cost of the raw materials. Its designed to run on glucose, the type of sugar produced from corn or sugarcane. But the company is adapting it to work with sugars from non-food sources such as wood chips, which include glucose but also other sugars such as xylose.

Audis partnership with Global Bioenergies is part of push by the automaker to reduce greenhouse gas emissions in the face of tightening regulations. Audi recently announced two other investments in cleaner fuels. It funded a project to make methane using renewable energythe methane can be used to run Audis natural-gas fueled cars (see Audi to Make Fuel Using Solar Power). And it funded Joule Unlimited, which is using photosynthetic microrganisms to make ethanol and diesel (see Audi Backs a Biofuels Startup).

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Audi Bets on Bio Gasoline Startup

GENETICS (But Actually A Rant )
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Genetics- Non-Mendelian Inheritance

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Genetics Screencast

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Genetics Screencast - Video

27th Anniversary Sports Spectacular Benefiting Cedars-Sinai Medical Genetics Institute - Red Carpet
New site http://ginacarano.meximas.com/ This channel is dedicated to one of the most prominent figures in the world of martial arts, won the tournament in Th...

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A guide to herd genetics reports - brought to you by DairyCo
A short presentation on herd genetics reports, how to register and using the reports to evaluate different breeds.

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Utopia - S2E17 - Down the Rabbit Hole - Return to Sunshine Genetics Lab
Welcome to the second season of the Utopia Server. Come watch as we check out all that modded Minecraft 1.6.4 has to offer. Our server, our modpack, our rul...

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Human Genome1 Gene Therapy

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25 Jan 2014 00:01

PROFESSOR Sir Ken Murray, one of the first researchers in genetic engineering, saved many lives worldwide.

PRESSTEAM

A SCIENTIST who developed the first vaccine for hepatitis B has left 30million to the charity he founded in Edinburgh.

Professor Sir Ken Murrays groundbreaking work was credited with saving lives worldwide.

The 82-year-old, who worked at Edinburgh University for more than 30 years and was one of the first researchers in genetic engineering, died at home in the city last April.

His will reveals his estate was worth 45million with his fortune built on royalties from the vaccine.

The main beneficiaries are the Darwin Trust of Edinburgh, founded by Sir Ken in 1983.

They will receive 30million to support the education of young scientists and fund research and facilities at Edinburgh University.

Trust chairman Dr John Tooze, 75, said: Ken was an extraordinary man who remained very modest despite the huge royalties that his hepatitis B vaccine brought him.

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Man who developed first vaccine for hepatitis B leaves £30million to charity

Bullwinkle Whitetail Deer , Disease?..Genetics? New breed?Unknown.
Bullwinkle Deer!!!!!!!!!!!!!!!!!!!! I could not believe my eyes this morning when I came across this in the Local Newspaper in the hunting section. Bullwinkl...

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The counseling that goes along with testing for breast cancer risk genes can be delivered just as effectively over the phone as in person, and at lower cost, according to new research.

Researchers found that women counseled over the phone before testing fared about the same in their understanding of the test and reactions to it as those counseled in person.

"Genetic counselors are not evenly distributed across the country," Marc Schwartz said. "So there are parts of the country that don't have easy access to genetic cancer counseling."

Schwartz is the study's lead author. He's also co-leader of the Cancer Prevention and Control Program and co-director of the Fisher Center for Familial Cancer Research at the Georgetown Lombardi Comprehensive Cancer Center in Washington, D.C.

Genetic counseling is a way to provide information and support to people and families who have genetic disorders or may be at risk for inherited conditions, according to the U.S. National Institutes of Health.

"We have - by virtue of the incredibly high demand for genetic risk counseling - been looking for alternative avenues," Dr. Sofia Merajver told Reuters Health.

Merajver is director of the Breast and Ovarian Cancer Risk and Evaluation Program at the University of Michigan Health System's Comprehensive Cancer Center in Ann Arbor. She was not involved with the new study.

"We've all done telephone counseling for patients who are very far away, very ill and for patients who couldn't afford it," she said.

Telephone counseling is not necessarily encouraged, however. Schwartz also said it has been somewhat controversial.

For the new study, researchers from Washington, New York, Boston and Burlington, Vermont, recruited 669 women who were between the ages of 21 and 85 years old.

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Telephone genetic counseling good for breast cancer risk ...

When the FDA shut down biotech company 23andMes direct-to-consumer Personal Genome Service, many in the biotech community worried that the FDA was risking the survival of a nascent industry that would revolutionize medical care with new genetic technologies. But lost in the story about 23andMe was the news that the FDA had just issued its very first approval for a next-generation DNA analysis machine, clearly signaling that the agency recognizes the growing significance of genetic testing in medicine.

The FDA approved a machine called the MiSeqDx, the first FDA-regulated test system that allows laboratories to develop and validate sequencing of any part of a patients genome. For a few hundred dollars, this little machine can analyze an amount of DNA in 24 hours that, 10 years ago, would have taken a dozen machines two weeks and cost nearly a million dollars. The rate of improvement in our technical capacity to read out DNA has been stunningly rapid, outpacing even the expectations of Moores Law, the gold standard for progress in the computer industry. Biomedical researchers can now consider ways to bring DNA analysis into the clinic that would have been impossible before. Rather than testing for mutations in one gene at a time, and in only very specific groups of patients, its becoming feasible to scan much larger portions of a patients DNA as part of a routine health assessment for everyone. Whats happening in medical genetics is the equivalent of having the operating costs of a private jet suddenly drop to those of a Honda Civicin which case youd probably start considering previously unthinkable destinations for not only the annual family vacation, but for three-day weekends as well.

In a report issued last fall (PDF), the FDA laid out its view of the regulatory challenges posed by personalized genetic medicine. At issue is the idea that with cheap, accessible DNA analysis, medical care can be personalized to match each persons genetic makeup. Cancer diagnoses, rather than being based on abnormalities that are visible under a microscope, would instead be classified more effectively by their underlying genetic mutations. The typical trial-and-error approach to find the right drug for a patient suffering from depression would give way to a genetic test that would indicate the best drug.

It sounds great in principle, but the result is a major headache for the FDA, because modern genetic medicine is demolishing regulatory concepts and categories that the agency has long used to ensure that drugs and diagnostics are safe and effective.

Take genetic tests for instance. The two primary criteria the FDA uses to evaluate diagnostics are analytical validity and clinical validity. As the FDA report describes them, analytical validity refers to how well the test measures what it is supposed to measure, whereas clinical validity looks at how well the test predicts who has or does not have a disease or condition for which it is being tested. A typical diagnostic test is required to be both analytically and clinically valid, but for large-scale genetic tests this doesnt make sense. Tests that analyze hundreds or thousands of regions of your DNA at once can be analytically validthat is, they accurately determine the identity of mutations in your genomebut the clinical validity will vary with the individual mutation, depending on whether that mutation has a discernible effect. Furthermore, clinical validity for any one mutation will often be in flux, as new research clarifies the role of poorly understood mutations.

Another challenge with personalization is that a drug and a diagnostic test are more likely to be paired in their development. Some drugs, like the cystic fibrosis drug Kalydeco, are deliberately targeted only at patients with a specific mutation. Pairing genetic tests with therapeutics makes it difficult to track down the source of problems when something doesnt work. As the FDA report notes, An adverse event associated with the use of a therapeutic product may have arisen as a result of failure of the test to identify the optimal subset of patients due to design deficiencies, manufacturing deficiencies, or operator error.

To respond to these challenges, the FDA report describes the changes youd expect from a large and complex government agency tasked with keeping up with a large and complex industry. There are restructurings, efforts to increase communication and coordination among different agency centers, and committees to rethink the process by which some new treatments and diagnostics are approved.

WHILE THE FDA MAY be making an admirable effort to confront the issues raised in the report, the authors ignored the elephant in the room: the wild frontier of direct-to-consumer genetic testing, represented by 23andMe. Consumer-oriented genome services undermine one of the biggest regulatory concepts that the FDA depends on: whether or not something counts as a medical diagnostic device. Does the FDA need to protect you from information about your genome, especially if some of that information is potentially unreliable? Or should anyone be able to buy a tentative analysis of their genome based on the latest research?

This is where medical genetics shades into recreational genetics. It doesnt help that these services are often advertised with the dubious claim that they will empower you to take your health into your own hands. As a group of researchers, physicians, and health policy experts noted in a recent commentary, there is little evidence to support the basic premise implied by the empowerment rhetoricnamely that individuals will use genomic risk information to adopt a healthier lifestyle and, thus, reduce their risk for chronic diseases.

Even if a personal genome analysis is not useful yet, it is hard to make the case that we should be barred from it. These services feed our curiosity about ourselves, and they are an opportunity to educate consumers about genetics. Of course our genetics are inextricably tied up with our health, which means that direct-to-consumer genetic services will always threaten to impinge on the FDAs territory. How it should respond is an issue not yet resolved.

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CGS : The FDA Is Not Anti-Genetics

A New Horizon - Episode 9: Advanced Genetics
Hello and welcome. I #39;m mallrat208 and you #39;re watching #39;A New Horizon #39; my #39;Feed the Beast: Horizons #39; Let #39;s Play/Discovery. Today I dive headfirst into the Adv...

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