1. Gene Therapy

Category Archives: Gene Therapy

'Bubble boy' progress reported

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From left: Tushar Menon, Inder Verma and Amy Firth. Salk Institute

From left: Tushar Menon, Inder Verma and Amy Firth. / Salk Institute

Those born with the immune disorder SCID-X1, or "bubble boy disease" may one day benefit from a new treatment to give them a functioning immune system, if new research from the Salk Institute succeeds.

Working with cultures of induced pluripotent stem cells from a patient, Salk scientists led by gene therapy expert Inder Verma repaired the genetic defect that causes the disease. Infants born with this inherited condition have virtually no immune resistance, and can be killed by infections easily defeated by normal immune systems.

Researchers were able to generate what appear to be mature NK, or "natural killer" immune cells, the first time this has been done. They also generated progenitors of T cells. This doesn't repair all the immune system, but it's a big step in that direction.

These preliminary results may pave the way to an alternative from treating these patients, Verma said. At present, patients can be treated with bone marrow transplants, but matching donors are hard to find. Gene therapy using a viral vector to repair the defect has been successful, but has caused leukemia in some patients when the corrective gene went into the wrong place. Newer forms of this therapy appear to have reduced the risk, but long-term followups of those treated are still in progress.

Salk researchers dispensed with viruses entirely by using the TALEN technology, which allows genetic editing without viruses, and is also more precise.

The study was published in the journal Cell Stem Cell on March 12. Tushar Menon and Amy L. Firth are the first authors. Verma is the senior author.

SCID-X1 is caused by an inactivating mutation on a gene called IL-2Rg located on the X chromosome, which means it exclusively affects males. (For females who carry the mutation on one chromosome, the functional gene on the other chromosome suffices).

One-letter mutation

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'Bubble boy' progress reported

New Discovery Moves Gene Editing Closer to Use in Humans

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The gene editing method called CRISPR is already used in the lab to insert and remove genome defects in animal embryos

Genome editing has generated controversy, with unconfirmed reports of its use in human embryos. Credit: NIAID/Flickr

A tweak to a technique that edits DNA with pinpoint precision has boosted its ability to correct defective genes in people. Called CRISPR, the method is already used in the lab to insert and remove genome defects in animal embryos. But the genetic instructions for the machinery on which CRISPR reliesa gene-editing enzyme called Cas9 and RNA molecules that guide it to its targetare simply too large to be efficiently ferried into most of the human bodys cells.

This week, researchers report a possible way around that obstacle: a Cas9 enzyme that is encoded by a gene about three-quarters the size of the one currently used. The finding, published on 1April inNature, could open the door to new treatments for a host of genetic maladies (F. A. Ranetal. Naturehttp://dx.doi.org/10.1038/nature14299; 2015).

There are thousands of diseases in humans associated with specific genetic changes, says David Liu, a chemical biologist at Harvard University in Cambridge, Massachusetts, who was not involved in the latest study. A fairly large fraction of those have the potential to be addressed using genome editing.

Genome editing has generated controversy, with unconfirmed reports of its use in human embryos. Some scientists have expressed concern that the technique might be used by fertility doctors to edit the genes of human embryos before its safety is established (see alsoE.Lanphieret al. Nature519,410411; 2015). That concern is exacerbated by the fact that changes made by the procedure in embryos would be passed to all subsequent generations without giving anyone affected the opportunity to consent (seeNature519,272; 2015). But in the non-reproductive cells of children and adults, where intergenerational issues are not a concern, researchers and companies are already racing to develop CRISPR as a clinical tool.

The ethics of that pursuit may be more straightforward, but its execution can be harder than using CRISPR in embryos. An embryo consists of a small number of cells that give rise to a human. To edit the genome at that stage is simply a matter of injecting the necessary CRISPR components into a few cells. An adult human, however, is a mix of trillions of cells assembled into many different tissues. Researchers fret over how to target the CRISPR machinery to the specific cells where defective genes are disrupting physiological processes.

You can have the most optimal gene-editing system in the world, but if you cant deliver it to the proper cell type, its irrelevant, says Nessan Bermingham, chief executive of Intellia Therapeutics in Cambridge, Massachusetts, which aims to bring genome editing to the clinic. Were spending a tremendous amount of time working on it.

Snug fit Gene-therapy researchers often harness a virus called AAV to shuttle foreign genes into mature human cells. However, most laboratories use a gene encoding the Cas9 protein that is too large to fit in the snug confines of the AAV genome alongside the extra sequences necessary for Cas9 function.

Feng Zhang of the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, and his colleagues decided to raid bacterial genomes for a solution, because the CRISPR system is derived from a process that bacteria use to snip unwanted DNA sequences out of their genomes. Zhangs team analysed genes encoding more than 600 Cas9 enzymes from hundreds of bacteria in search of a smaller version that could be packaged in AAV and delivered to mature cells.

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New Discovery Moves Gene Editing Closer to Use in Humans

Mini enzyme moves gene editing closer to the clinic

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SPL

The bacterium Staphylococcus aureus is host to a smaller version of the enzyme used in the CRISPR technique for gene editing.

A tweak to a technique that edits DNA with pinpoint precision has boosted its ability to correct defective genes in people. Called CRISPR, the method is already used in the lab to insert and remove genome defects in animal embryos. But the genetic instructions for the machinery on which CRISPR relies a gene-editing enzyme called Cas9 and RNA molecules that guide it to its target are simply too large to be efficiently ferried into most of the human bodys cells.

This week, researchers report a possible way around that obstacle: a Cas9 enzyme that is encoded by a gene about three-quarters the size of the one currently used. The finding, published on 1April in Nature, could open the door to new treatments for a host of genetic maladies (F. A. Ran etal. Nature http://dx.doi.org/10.1038/nature14299; 2015).

There are thousands of diseases in humans associated with specific genetic changes, says David Liu, a chemical biologist at Harvard University in Cambridge, Massachusetts, who was not involved in the latest study. A fairly large fraction of those have the potential to be addressed using genome editing.

Genome editing has generated controversy, with unconfirmed reports of its use in human embryos. Some scientists have expressed concern that the technique might be used by fertility doctors to edit the genes of human embryos before its safety is established (see also E.Lanphier et al. Nature 519, 410411; 2015). That concern is exacerbated by the fact that changes made by the procedure in embryos would be passed to all subsequent generations without giving anyone affected the opportunity to consent (see Nature 519, 272; 2015). But in the non-reproductive cells of children and adults, where intergenerational issues are not a concern, researchers and companies are already racing to develop CRISPR as a clinical tool.

The ethics of that pursuit may be more straightforward, but its execution can be harder than using CRISPR in embryos. An embryo consists of a small number of cells that give rise to a human. To edit the genome at that stage is simply a matter of injecting the necessary CRISPR components into a few cells. An adult human, however, is a mix of trillions of cells assembled into many different tissues. Researchers fret over how to target the CRISPR machinery to the specific cells where defective genes are disrupting physiological processes.

You can have the most optimal gene-editing system in the world, but if you cant deliver it to the proper cell type, its irrelevant, says Nessan Bermingham, chief executive of Intellia Therapeutics in Cambridge, Massachusetts, which aims to bring genome editing to the clinic. Were spending a tremendous amount of time working on it.

Gene-therapy researchers often harness a virus called AAV to shuttle foreign genes into mature human cells. However, most laboratories use a gene encoding the Cas9 protein that is too large to fit in the snug confines of the AAV genome alongside the extra sequences necessary for Cas9 function.

Feng Zhang of the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, and his colleagues decided to raid bacterial genomes for a solution, because the CRISPR system is derived from a process that bacteria use to snip unwanted DNA sequences out of their genomes. Zhangs team analysed genes encoding more than 600 Cas9 enzymes from hundreds of bacteria in search of a smaller version that could be packaged in AAV and delivered to mature cells.

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Mini enzyme moves gene editing closer to the clinic

Biotech's next buzz: Gene therapy?

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Imagine if instead of using drugs or surgery, a doctor could simply insert a gene into a patient's cell to treat or prevent disease.

Gene therapy is endeavoring to do just that and could emerge in the coming years as a viable alternative for treatment. Certain conditions that previously couldn't be treated, such as forms of blindness, types of cancer or more rare conditions like sickle cell disease are now being researched with this alternative treatment.

Phil Nadeau, biotechnology analyst with Cowen and Company, predicts there will be several gene therapy products approved by the F.D.A. within the next few years. He told CNBC recently he sees more than $1 billion in gene therapy sales worldwide, which ultimately means significant investments in gene therapy in the near future.

"In the past, most major companies stayed away from having gene therapy programs. In the future, we think, it's going to be a standard treatment," he said.

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Biotech's next buzz: Gene therapy?

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological… – Video

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Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological...
Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy. Alessandra Ricca et al (2015), Human...

By: KeSimpulan

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Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological... - Video

Cancer gene therapy biotech MultiVir files for a $70 million IPO

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MultiVir, a biotech developing gene therapies for cancer, filed on Monday with the SEC to raise up to $70 million in an initial public offering.

The Houston, TX-based company, which was founded in 2009, plans to list on the NASDAQ under the symbol MVIR. MultiVir initially filed confidentially on December 22, 2014. RBC Capital Markets is the sole bookrunner on the deal. No pricing terms were disclosed.

Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital's research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital, the Renaissance IPO ETF (symbol: IPO) or the Global IPO Fund (symbol: IPOSX) , may have investments in securities of companies mentioned.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

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Cancer gene therapy biotech MultiVir files for a $70 million IPO

Celladon Heart-Failure Study Looms Large as Next Big Test for Gene Therapy

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NEW YORK (TheStreet) -- The next investor referendum on the resurgent gene-therapy field will arrive next month whenCelladon (CLDN)is expected to announce results from a mid-stage study of a gene therapy aimed atimproving the heart's pumping ability in patients suffering fromthe organ's advanced failure.

Gene therapy uses engineered viruses to replace defective, disease-causing genes. Celladon's lead therapy, Mydicar, is a virus engineered to insert a working gene capable of producing a protein called SERCA2a into heart-failure patients. SERCA2a is responsible for helping heart muscles contract and pump blood more efficiently. Heart-failure patients have low levels of SERCA2a and hearts that do a poor job pumping blood around the body. Celladon believes infusing Mydicar should lead to higher SERCA2a levels and improved heart function.

Must Read: 5 Stocks Warren Buffett Is Selling

Celladon went public in January 2014 at $8 per share. The stock was tradingat $24.10, down 1.8%, on Wednesday morning, after rising by more than 30% in March as investors anticipate the Mydicar study results.

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Celladon Heart-Failure Study Looms Large as Next Big Test for Gene Therapy

University And Biotech Firm Team Up On Colorblindness Therapy

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A simulation from the Neitz lab of what colorblindness looks like, with normal color vision on the left and red-green colorblindness on the right. Courtesy of Neitz Laboratory hide caption

A simulation from the Neitz lab of what colorblindness looks like, with normal color vision on the left and red-green colorblindness on the right.

More than 10 million Americans have trouble distinguishing red from green or blue from yellow, and there's no treatment for colorblindness.

A biotech company and two scientists hope to change that.

On Wednesday, Avalanche Biotechnologies in Menlo Park and the University of Washington in Seattle announced a licensing agreement to develop the first treatment for colorblindness. The deal brings together a gene therapy technique developed by Avalanche with the expertise of vision researchers at the University of Washington.

"Our goal is to be treating colorblindness in clinical trials in patients in the next one to two years," says Thomas Chalberg, the founder and CEO of Avalanche.

Dalton the squirrel monkey during the color vision test. Courtesy of Neitz Laboratory hide caption

Dalton the squirrel monkey during the color vision test.

The agreement has its roots in a scientific breakthrough that occurred six years ago. That's when two vision researchers at the University of Washington used gene therapy to cure a common form of colorblindness in squirrel monkeys.

"This opened the possibility of ultimately getting this to cure colorblindness in humans," says Jay Neitz, who runs the Color Vision Lab at UW along with his wife, Maureen Neitz.

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University And Biotech Firm Team Up On Colorblindness Therapy

Lumber Liquidators Scandal with Dr Mitch Gaynor on Fox Business – Video

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Lumber Liquidators Scandal with Dr Mitch Gaynor on Fox Business
Subscribe to the GeneChanger YouTube: http://goo.gl/jOPcep Dr Mitch Gaynor, Medical Oncologist at Cornell Medical Center Author of "Gene Therapy Plan" on Fox Business. The Gene Therapy...

By: Mitchell Gaynor M.D.

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Lumber Liquidators Scandal with Dr Mitch Gaynor on Fox Business - Video


  1. Gene Therapy