Explaining gene therapy for wet AMD
Shows how wet macular degeneration destructs vision, how secretion gene therapy works and how people can benefit.

By: Elizabeth Rakoczy

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Explaining gene therapy for wet AMD - Video

The Alpha Clinic for Cell Therapy and Innovation | City of Hope
A new grant to City of Hope from the California Institute for Regenerative Medicine (CIRM) will make it possible for novel stem cell based therapies developed here at City of Hope to...

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The Alpha Clinic for Cell Therapy and Innovation | City of Hope - Video

Nobel Prize winners raise alarm over genetic engineering of humans.

A group of senior American scientists and ethics experts is calling for debate on the gene-engineering of humans, warning that technology able to change the DNA of future generations is now imminent.

In policy recommendations published today in the journal Science, eighteen researchers, including two Nobel Prize winners, say scientists should accept a self-imposed moratorium on any attempt to create genetically altered children until the safety and medical reasons for such a step can be better understood.

The concern is over a rapidly advancing gene-editing technology, called CRISPR-Cas9, which is giving scientists the ability to easily alter the genome of living cells and animals (see Genome Surgery). The same technology could let scientists correct DNA letters in a human embryo or egg cell, for instance to create children free of certain disease-causing genes, or perhaps with improved genetics.

What we are trying to do is to alert people to the fact that this is now easy, says David Baltimore, a Nobel Prize winner and former president of Caltech, and an author of the letter. We cant use the cover we did previously, which is that it was so difficult that no one was going to do it.

Many countries already ban germ line engineeringor changing genes in a way that would be heritable from one generation to the nextonethical or safety grounds. Others, like the U.S., have strict regulations that would delay the creation of gene-edited children for years, if not decades. But some countries have weak rules, or none at all, and Baltimore said a reason scientists were speaking publicly now was to keep people from doing anything crazy.

The advent of CRISPR is raising social questions of a kind not confronted since the 1970s, when the ability to change DNA in microrganisms was first developed. In a now famous meeting in 1975, in Asilomar, California, researchers agreed to avoid certain kinds of experiments that were then deemed dangerous. Baltimore, who was one of the organizers of the Asilomar meeting, says the scientists behind the letter want to offer similar guidance for gene-engineered babies.

The prospect of genetically modified humans is surprisingly close at hand. A year ago, Chinese researchers created monkeys whose DNA was edited using CRISPR (see 10 Breakthrough Technologies 2014: Genome Editing).

Since then,several teams of researchers in China, the U.S., and the U.K. have begun using CRISPR to change the DNA of human embryos, eggs, and sperm cells, with an eye toward applying the technology at in vitro fertility (IVF) clinics. That laboratory research was described by MIT Technology Review earlier this month (see Engineering the Perfect Baby).

Last week, in Nature, representatives of an industry group, the Alliance for Regenerative Medicine, recommended a wider moratorium that would also include a cessation of such laboratory studies, which it termed dangerous and ethically unacceptable (see Industry Body Calls for Gene-Editing Moratorium).

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Scientists Call for a Summit on Gene-Edited Babies

Gene Therapy Respiratory Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Respiratory Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape provides in depth insights into the Respiratory gene therapy. It has covered 5+...

By: James Jacob

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Gene Therapy Respiratory Insight: Trends and Challenges Analysed in Research Report - Video

Gene Therapy, Ex-Vivo and In-Vivo

By: Pedro Loureno

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Gene Therapy, Ex-Vivo and In-Vivo - Video

Science Advances : Gene therapy rescues disease phenotype in a spinal muscular atrophy......
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model. Monica Nizzardo et al (2015), Scie...

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Science Advances : Gene therapy rescues disease phenotype in a spinal muscular atrophy...... - Video

Gene Therapy Gastrointestinal Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Gastrointestinal Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy ...

By: James Jacob

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Gene Therapy Gastrointestinal Insight: Trends and Challenges Analysed in Research Report - Video

Stamford, CT (PRWEB) March 16, 2015

Alliance for Cancer Gene Therapy (ACGT) the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer has achieved a major milestone, surpassing $25 million donated to innovative and breakthrough cancer research. ACGT was founded by Barbara Netter and her late husband, Edward, in 2001 with the goal of transforming cancer into a manageable, treatable disease.

Pushing the foundation across the $25 million threshold are a pair of three-year, $250,000 grants to two esteemed scientists: Meenakshi Hegde, MD, of Texas Childrens Cancer Center at Baylor College of Medicine in Houston, TX, and Christopher Jewell, PhD, at University of Maryland, College Park. Dr. Hegdes work will focus on immunotherapy, specifically adoptive cellular therapy for melanoma. Dr. Jewells research is centered on harnessing intra-lymph node gene therapy to promote tumor immunity. The grantees will develop genetically-modified T cells and cancer vaccines with the potential to stop cancer in its tracks.

Drs. Hegde and Jewell are two outstanding scientists in the vanguard of treating and defeating cancer, said Barbara Netter, ACGTs President. Their work offers tremendous hope to those battling cancer, and also to their loved ones.

ACGT grants are awarded to promising researchers whose work dovetails with the foundations mission: Leveraging cell and gene therapies to supplant the more harrowing cancer treatments like radiation, chemotherapy and surgery. ACGTs $25 million in grants have funded watershed research and trials such as those that activate patients own immune systems to battle cancer cells. These trials have saved the lives of cancer patients otherwise believed to be beyond treatment.

The two most recent grants continue ACGTs mission of equipping innovative scientists with the tools and support to revolutionize the fight against cancer. ACGT grants range from $250,000 to $1 million, and reward both young, promising researchers and their more established colleagues. Past recipients include such pre-eminent scientists as University of Pennsylvanias Dr. Carl June and Memorial Sloan-Ketterings Dr. Michel Sadelain; this past summer, the Food and Drug Administration (FDA) granted breakthrough status to immunotherapy treatments for leukemia developed by each of these scientists for which ACGT provided early funding.

About Alliance for Cancer Gene Therapy (ACGT) Established in 2001, ACGT (http://www.acgtfoundation.org) is the nations only not-for-profit dedicated exclusively to cell and gene therapy treatments for all types of cancer. One-hundred percent of contributions go directly to research. ACGT has funded 46 grants in the U.S. and Canada since its founding in 2001 by Barbara Netter, President, and her late husband, Edward, to conduct and accelerate critically needed innovative research. Since its inception, ACGT has awarded 31 grants to Young Investigators and 15 grants to Clinical Investigators, totaling more than $25 million in funding. ACGT is located at 96 Cummings Point Road, Stamford, CT 06902.

ACGT on Facebook: http://www.facebook.com/ACGTfoundation ACGT on Twitter: http://www.twitter.com/ACGTfoundation ACGT on YouTube: http://www.youtube.com/user/ACGTfoundation

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ACGT Surpasses $25 Million Funding Milestone with Two New Grants

Gene Therapy, Viral

By: Pedro Loureno

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Gene Therapy, Viral - Video

Gene Therapy Oncology Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Oncology Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy profiles core.

By: James Jacob

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Gene Therapy Oncology Insight: Trends and Challenges Analysed in Research Report - Video

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. Thus far, the new therapy has proven safe.

"The result was stunning," said Timothy Nichols, MD, director of the Francis Owen Blood Research Laboratory at the University of North Carolina School of Medicine and co-senior author of the paper. "Just a small amount of new factor IX necessary for proper clotting produced a major reduction in bleeding events. It was extraordinarily powerful."

The idea behind gene therapy is that doctors could give hemophilia patients a one-time dose of new clotting genes instead of a lifetime of multiple injections of recombinant factor IX that until very recently had to be given several times a week. A new FDA-approved hemophilia treatment lasts longer than a few days but patients still require injections at least once or twice a month indefinitely.

This new gene therapy approach, like other gene therapy approaches, would involve a single injection and could potentially save money while providing a long-term solution to a life-long condition. A major potential advantage of this new gene therapy approach is that it uses lentiviral vectors, to which most people do not have antibodies that would reject the vectors and make the therapy less effective.

In human clinical studies, approximately 40 percent of the potential participants screened for a different kind of viral vector -- called adeno-associated viral vectors -- have antibodies that preclude them from entering AAV trials for hemophilia gene therapy treatment. This means that more people could potentially benefit from the lentivirus gene therapy approach.

Hemophilia is a bleeding disorder in which people lack a clotting factor, which means they bleed much more easily than people without the disease. Often, people with hemophilia bleed spontaneously into joints, which can be extremely painful and crippling. Spontaneous bleeds into soft tissues are also common and can be fatal if not treated promptly. Hemophilia A affects about one in 5,000 male births. These patients do not produce enough factor VIII in the liver. This leads to an inability to clot. Hemophilia B affects about one in 35,000 births; these patients lack factor IX.

This new method was spearheaded by Luigi Naldini, PhD, director of the San Raffaele Telethon Institute for Gene Therapy and co-senior author on the Science Translational Medicine paper.

For this study, Naldini and Nichols developed a way to use a lentivirus, which is a large retrovirus, to deliver factor IX genes to the livers of three dogs that have naturally occurring hemophilia. The researchers removed the genes involved in viral replication. "Essentially, this molecular engineering rendered the virus inert," Nichols said. "It had the ability to get into the body but not cause disease." This process turned the virus into a vector -- simply a vehicle to carry genetic cargo.

Unlike some other viral vectors that have been used for gene therapy experiments, the lentiviral vector is so large that it can carry a lot of payload -- namely, the factor IX genes that people with hemophilia B lack. (This approach could also be used for hemophilia A where the FVIII gene is considerably larger.)

These viral vectors were then injected directly into the liver or intravenously. After more than three years, the three dogs in the study experienced zero or one serious bleeding event each year. Before the therapy, the dogs experienced an average of five spontaneous bleeding events that required clinical treatment. Importantly, the researchers detected no harmful effects.

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New gene therapy for hemophilia shows potential as safe treatment

Using mutant protein, CHOP hematologist safely removes unwanted antibodies, reverses hemophilia in dog model of bleeding disease

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. In addition, the "turbocharged" clotting factor protein eliminated pre-existing antibodies that often weaken conventional treatments for people with hemophilia.

"Our findings may provide a new approach to gene therapy for hemophilia and perhaps other genetic diseases that have similar complications from inhibiting antibodies," said the study leader, Valder R. Arruda, M.D., Ph.D., a hematology researcher at The Children's Hospital of Philadelphia (CHOP).

Arruda and colleagues published their animal study results in the print edition of Blood on March 5.

Hemophilia is an inherited bleeding disorder that famously affected European royal families descended from Queen Victoria. Most commonly occurring in two types, hemophilia A and hemophilia B, the disease impairs the blood's ability to clot, sometimes fatally. When not fatal, severe hemophilia causes painful, often disabling internal bleeding and joint damage.

Doctors treat hemophilia with frequent intravenous infusions of blood clotting proteins called clotting factors, but these treatments are expensive and time-consuming. Moreover, some patients develop inhibiting antibodies that negate the effectiveness of the infusions.

For more than two decades, many research teams, including at CHOP, have investigated gene therapy strategies that deliver DNA sequences carrying genetic code to produce clotting factor in patients. However, this approach has been frustrated by the body's immune response against vectors--the non-disease-causing viruses used to carry the DNA. Those responses, which defeated initial benefits seen in experimental human gene therapy, were dose-dependent: higher amounts of vectors caused more powerful immune responses.

Arruda and colleagues therefore investigated gene therapy that used lower dosages of vector (adeno-associated viral-8 vector, or AAV-8 vector) to produce a more potent clotting factor--a variant protein called FIX-Padua.

Arruda was part of a scientific team in 2009 that discovered FIX-Padua in a young Italian man who had thrombosis, excessive clotting that can dangerously obstruct blood vessels. A mutation produced the mutant clotting factor, called FIX-Padua, named after the patient's city of residence. This was the first mutation in the factor IX gene found to cause thrombosis. All previously discovered FIX mutations lead to hemophilia, the opposite of thrombosis.

FIX-Padua is hyperfunctional--it clots blood 8 to 12 times more strongly than normal, wild-type factor IX. In the current study, the researchers thus needed to strike a balance--to relieve severe hemophilia in dogs, by using a dose strong enough to allow clotting, but not enough to cause thrombosis or stimulate immune reactions. "Our ultimate goal is to translate this approach to humans," said Arruda, "by adapting this variant protein found in one patient to benefit other patients with the opposite disease."

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Study bolsters 'turbocharged' protein as a promising tool in hemophilia gene therapy

Gene-editing companies say research on altering the DNA of human reproductive cells is dangerous and unethical.

Officials of a biotechnology industry group have called for a voluntary moratorium on using new DNA-editing techniques to change the genetic characteristics of human embryos in laboratory research.

In an editorial published today by the journal Nature, Edward Lanphier, CEO of the biotechnology company Sangamo Biosciences, and four colleagues write that scientists should agree not to modify the DNA of human reproductive cells because it raises safety and ethical risks including the danger of unpredictable effects on future generations.

New gene-editing techniques, in particular one called CRISPR, have given scientists powerful and useful new ways to swap and change DNA letters inside of living cells for the first time (see Genome Surgery).

Recently, some scientific teams have started to study whether CRISPR would be able to correct disease genes in future generations of peoplefor instance, by repairing genes during in vitro fertilization, or in eggs or sperm. The idea of such germ line modification would be to install healthy versions of genes, which children would be born with.

The emergence of active research around germ-line editing, which is taking place in China, at Harvard University, and at a publicly traded biotechnology company called OvaScience, were described last week by MIT Technology Review (see Engineering the Perfect Baby).

But the idea of using editing technology to improve children is as controversial as it is medically powerful. In their editorial, Lanphier, whose coauthors include Fyodor Urnov, co-developer of a different gene-editing system, raise the concern that such techniques might be exploited for non-therapeutic modifications. That could mean, for instance, changing the physical traits of children.

The availability of technology to carry out genetic engineering in human germ-line cells is driving intense debate in scientific circles and may eventually become a legal issue in the United States and other countries.

The authors call for a cessation of basic research is unusual and likely to be opposed by scientists as an intrusion on the quest for scientific knowledge.

George Church, a professor at Harvard Medical School whose laboratory studies CRISPR and germ-line editing, says a voluntary moratorium would be weak compared with existing regulations that nearly all countries impose on the use of new medical technologies until they are proven safe and effective in animals or human [tests]. Church was referring to rules governing the birth of actual gene-edited children, not basic research.

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Industry Body Calls for Gene-Editing Moratorium

FRESNO, Calif. (KFSN) --

Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers."

Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching."

Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations.

"It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30.

Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope.

University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision.

"It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30.

Boye says the therapy has worked in animals. "We're able to show, via what's called an electra retinal gram, that the retinal function has been restored to the mice," she explained.

Gene therapy is still an investigational treatment with risks and only available for those in a clinical trial. Right now there are hundreds of studies underway to treat conditions like LCA, cancer and HIV. It's hope that one day Andy could put down his cane and see his family for the first time.

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Gene therapy: Hope for the blind?

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities.

Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies.

In recent years however,researches have proposed a new method of battling the virus that involves gene therapy.

Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely.

In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV.

In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus.

The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.)

When HIV enters the body, it attacks specific immune cells. As the virus copies itself over and over, and kills more and more host cells, the immune system grows progressively weaker. If left untreated, this progressive weakening will give rise to AIDS.

In most cases, the HIV virus begins its attack by latching onto two separate protein structures on the surface of its target white blood cells. One of these structures is called CD4, and the other is called CCR5.

In the Nature study, researchers set out to engineer an antibody-like molecule that would mimic both of these proteins, so that it would act as decoy of sorts for the virus. Instead of latching onto a host cell, HIV would latch onto a specially enhanced protein molecule, or eCD4-Ig, that was released by the cell.

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With genetic engineering, scientists use decoy molecule to trick HIV

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities.

Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies.

In recent years however,researches have proposed a new method of battling the virus that involves gene therapy.

Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely.

In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV.

In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus.

The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.)

When HIV enters the body, it attacks specific immune cells. As the virus copies itself over and over, and kills more and more host cells, the immune system grows progressively weaker. If left untreated, this progressive weakening will give rise to AIDS.

In most cases, the HIV virus begins its attack by latching onto two separate protein structures on the surface of its target white blood cells. One of these structures is called CD4, and the other is called CCR5.

In the Nature study, researchers set out to engineer an antibody-like molecule that would mimic both of these proteins, so that it would act as decoy of sorts for the virus. Instead of latching onto a host cell, HIV would latch onto a specially enhanced protein molecule, or eCD4-Ig, that was released by the cell.

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A new twist on HIV vaccines shows results in monkeys, study says

Paying for gene therapy are annuities the next big thing
Paying for gene therapy are annuities the next big thing.

By: Reuters-BBC-Aljazeera

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Paying for gene therapy are annuities the next big thing - Video

Home Books Author Jeanne Ryan discusses Charisma and gene therapy

Author Jeanne Ryan talks with Hypable about her book Charisma and whether or not she would take her fictional drug if given the chance.

Charisma follows the story of Aislyn, a painfully shy junior in high school who suffers from social anxiety. Speaking up in class and trying to have fun with her friends at a party are well outside her comfort zone. She finds out about a new underground gene therapy treatment in the form of a drug called Charisma. Aislyns personality completely changes, and suddenly shes exactly the person she wanted to be all along.

But theres one problem. The therapy appears to be contagious and deadly. Worse yet, the doctor who gave her and a select number of other patients the drug suddenly disappears. Read our review.

Tell us 5 random facts about yourself.

Two of my brothers have the same name, kind of by accident. Just after my mom gave birth to her sixth kid in less than seven years, the nurse asked the name of her baby. Thinking the nurse meant the baby at home, my sleep-deprived mom gave her his name, and it ended up on the birth certificate.

I could swear that when I was a kid a certain soup company ran a commercial where the main lyric in its jingle was Constipation got you down? However, to this day, I have never been able to find anyone else who remembers this.

Until more recently than I care to admit, I thought narwhals were fictional. (In my defense, they look totally fake in photos!)

Id love to go to Burning Man one day.

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Author Jeanne Ryan discusses Charisma and gene therapy

S75RS04 Gene Therapy
Science Technology ITV Schools circa 1986.

By: Lammas Science

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S75RS04 Gene Therapy - Video

The Gene Therapy Plan: Taking Control of Your Genetic Destiny with Diet and Lifestyle
By Mitchell L. Gaynor MD (Author), Mehmet C. Oz M.D. (Foreword) http://genechanger.com/gene-therapy-plan-taking-control-genetic-destiny-diet-lifestyle/ From ...

By: Mitchell Gaynor M.D.

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The Gene Therapy Plan: Taking Control of Your Genetic Destiny with Diet and Lifestyle - Video