Category Archives: Gene therapy

By Ewen Callaway of Nature magazine

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Tales of biotechnology advances are unlikely to raise a lump in the throat or bring a tear to the eye on their own. Thats what makes Ricki Lewiss recent talk at Lexington Community Education so clever and engrossing. Lewis wrapped a lecture on human genetics and gene therapy in the genuinely moving stories of children whose lives were altered -- some for good, some for bad -- since the field was born in the early 1990s.

As in her book, The Forever Fix: Gene Therapy and the Boy Who Saved It, Lewis talked about the children impacted by rare diseases and the scientists who labored to get closer to a forever fix, a cure that would repair problems at the genetic level.

Lewis is the author of the science textbook Human Genetics: Concepts and Applications, now in its 10th edition and used worldwide, Discovery: Windows on the Life Sciences, and the novel Stem Cell Symphony, about using rock music to activate stem cells in the brain to cure disease. She holds a Ph.D in genetics from Indiana University, and is currently a genetic counselor and teaches at the Alden March Bioethics Institute of Albany Medical College.

She began the lecture with the story of 8-year-old Corey Haas, who in 2008, only four days after undergoing gene therapy to cure his hereditary blindness, screamed in pain when visible sunlight impacts his eyes for the first time.

Lewis uses Coreys story, and how he came to be one of the early beneficiaries of gene therapy, to tell the story of the birth of that miraculous treatment.

Corey was in the right place and the right time, she said. His blindness was treatable but the treatment was experimental.

Gene therapy involves the placement of a gene that a person cant make in their own body into virus cell which is implanted in the body. In Coreys case, his eyes were unable to use Vitamin A, which is necessary for the eye to take in and process light. A virus delivered DNA wrapped in a protein to correct faulty instructions in the affected body part, Lewis said.

Lewis also discussed the story of Jesse Gelsinger, a teenager whose death during gene therapy trials nearly brought the experimental treatment to a close while it was still in its infancy. While Gelsingers story was a tragic failure, Lewis said that scientists were committed to learning from their errors and moving the field further.

One of the most famous children Lewis discussed was David Vetter the so-called Bubble Boy of the 1970s, depicted in the film The Boy in the Plastic Bubble, starring John Travolta. Vetter died of an immunological disease in 1984 at age 30, but the condition is now treatable through gene therapy.

By treating rare diseases such as Vetters, Lewis explained that researchers will learn what they need to treat more common diseases in the future.

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Lewis speaks on gene therapy at Lexington Community Education event

A little more than three years ago a medical team from Berlin published the results of a unique experiment that astonished HIV researchers. The German group had taken bone marrow--the source of the body’s immune cells--from an anonymous donor whose genetic inheritance made him or her naturally resistant to HIV. Then the researchers transplanted the cells into a man with leukemia who had been HIV-positive for more than 10 years. Although treatment of the patient’s leukemia was the rationale for the bone marrow transplant therapy, the group also hoped that the transplant would provide enough HIV-resistant cells to control the man’s infection. The therapy exceeded the team’s expectations. Instead of just decreasing the amount of HIV in the patient’s blood, the transplant wiped out all detectable traces of the virus from his body, including in multiple tissues where it could have lain dormant. The German researchers were so surprised by the spectacularly positive results that they waited nearly two years before publishing their data.

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Antidepressants restore well-being to many people, but sometimes at the cost of such side effects as weight gain or loss of interest in sex. And these side effects can be just part of the frustration. As Robin Marantz Henig wrote in " Lifting the Black Cloud ," in the March issue of Scientific American , the drugs that have long dominated the market--the selective serotonin reuptake inhibitors (SSRIs) and the serotonin and norepinephrine reuptake inhibitors (SNRIs)--"do not help everyone and eventually fail in more than a third of users. A pill that seems to be working today might well stop helping tomorrow. And the drugs can take several weeks to start having a marked effect." Equally disturbing, some major pharmaceutical houses, such as GlaxoSmithKline , are pulling back from developing psychiatric medicines.

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A young woman who calls herself blue­berryoctopus had been taking anti­depressants for three years, mostly for anxiety and panic attacks, when she recounted her struggles with them on the Web site Experience Project. She said she had spent a year on Paxil, one of the popular SSRIs (selective serotonin reuptake inhibitors), but finally stopped because it destroyed her sex drive. She switched to Xanax, an ­antianxiety drug , which brought back her libido but at the cost of renewed symptoms. Then Paxil again, then Lexapro (another SSRI), then Pristiq, a member of a related class of antidepressants, the SNRIs (serotonin and norepinephrine reuptake inhibitors). At the time of the post, she was on yet another SSRI, Zoloft, plus Wellbutrin (a cousin of SNRIs that affects the activity of dopamine as well as norepinephrine), which was intended to counteract the sexual side effects of Zoloft. “I don’t notice much of a difference with the Wellbutrin, but I’m on the lowest dose now,” she wrote. “I’m going back to my psychiatrist next week, so maybe he’ll up it. Who knows.”

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Source:
http://rss.sciam.com/sciam/topic/gene-therapy

A little more than three years ago a medical team from Berlin published the results of a unique experiment that astonished HIV researchers. The German group had taken bone marrow--the source of the body’s immune cells--from an anonymous donor whose genetic inheritance made him or her naturally resistant to HIV. Then the researchers transplanted the cells into a man with leukemia who had been HIV-positive for more than 10 years. Although treatment of the patient’s leukemia was the rationale for the bone marrow transplant therapy, the group also hoped that the transplant would provide enough HIV-resistant cells to control the man’s infection. The therapy exceeded the team’s expectations. Instead of just decreasing the amount of HIV in the patient’s blood, the transplant wiped out all detectable traces of the virus from his body, including in multiple tissues where it could have lain dormant. The German researchers were so surprised by the spectacularly positive results that they waited nearly two years before publishing their data.

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

Antidepressants restore well-being to many people, but sometimes at the cost of such side effects as weight gain or loss of interest in sex. And these side effects can be just part of the frustration. As Robin Marantz Henig wrote in " Lifting the Black Cloud ," in the March issue of Scientific American , the drugs that have long dominated the market--the selective serotonin reuptake inhibitors (SSRIs) and the serotonin and norepinephrine reuptake inhibitors (SNRIs)--"do not help everyone and eventually fail in more than a third of users. A pill that seems to be working today might well stop helping tomorrow. And the drugs can take several weeks to start having a marked effect." Equally disturbing, some major pharmaceutical houses, such as GlaxoSmithKline , are pulling back from developing psychiatric medicines.

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

A young woman who calls herself blue­berryoctopus had been taking anti­depressants for three years, mostly for anxiety and panic attacks, when she recounted her struggles with them on the Web site Experience Project. She said she had spent a year on Paxil, one of the popular SSRIs (selective serotonin reuptake inhibitors), but finally stopped because it destroyed her sex drive. She switched to Xanax, an ­antianxiety drug , which brought back her libido but at the cost of renewed symptoms. Then Paxil again, then Lexapro (another SSRI), then Pristiq, a member of a related class of antidepressants, the SNRIs (serotonin and norepinephrine reuptake inhibitors). At the time of the post, she was on yet another SSRI, Zoloft, plus Wellbutrin (a cousin of SNRIs that affects the activity of dopamine as well as norepinephrine), which was intended to counteract the sexual side effects of Zoloft. “I don’t notice much of a difference with the Wellbutrin, but I’m on the lowest dose now,” she wrote. “I’m going back to my psychiatrist next week, so maybe he’ll up it. Who knows.”

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

Public release date: 6-Mar-2012 [ | E-mail | Share ]

Contact: Cody Mooneyhan cmooneyhan@faseb.org 301-634-7104 Federation of American Societies for Experimental Biology

Bethesda, MDA new research discovery by a team of Stanford and European scientists offers hope that people with atherosclerotic disease may one day be able to avoid limb amputation related to ischemia. A new research report appearing online in the FASEB Journal suggests that the delivery of genes for two molecules naturally produced by the body, called "PDGF-BB" and "VEGF" may successfully cause the body to grow new blood vessels that can save ischemic limbs.

"We hope that our findings will ultimately develop into a safe and effective therapy for the many patients, suffering from blocked arteries in the limbs, who are currently not adequately treated by surgery or drugs," said Helen M. Blau, Ph.D., a senior researcher involved in the work and Associate Editor of the FASEB Journal from the Baxter Laboratory for Stem Cell Biology at the Institute for Regenerative Medicine and Stem Cell Biology at Stanford. "This could help avoid the devastating consequences of limb amputations for both patients and their families."

To make this discovery, Blau and colleagues, including Andrea Banfi (now at Basel University), introduced the genes for PDGF-BB and VEGF into the muscles of mice, either independently or together. When high doses of VEGF alone were produced, they caused the growth of vascular tumors. When the two factors were produced in unbalanced amounts, tumor growth also occurred. When VEGF and PDGF were delivered in a fixed ratio relative to one another, however, no tumors occurred, and blood flow was restored to ischemic muscle tissue and damage repaired without any toxic effects. To achieve a "balanced" delivery of PDGF-BB and VEGF, scientists placed both genes in a single gene therapy delivery mechanism, called a "vector."

Although the report shows the feasibility of growing robust and safe new blood vessels that restore blood flow to diseased tissues, Blau points out that "there are multiple challenges to correcting peripheral vasculature disease by using proangiogenic gene therapy strategies. Two important challenges are what to deliver and how to get it to where it can have beneficial effects. Clinical success will require both delivering a gene therapy construct that encodes for effective angiogenic factors and ensuring that the sites of delivery are where the construct can have the greatest clinical benefit."

"This ingenious work, based on the latest techniques of molecular biology, tells us that it is possible to reinvigorate parts of our body that can't get enough blood to keep them going," said Gerald Weissmann, M.D., Editor-in-Chief of the FASEB Journal. "The next question is whether this approach will work in humans and exactly how to deliver the new treatment to places that need it the most."

###

Receive monthly highlights from the FASEB Journal by e-mail. Sign up at http://www.faseb.org/fjupdate.aspx. The FASEB Journal is published by the Federation of the American Societies for Experimental Biology (FASEB) and is the most cited biology journal worldwide according to the Institute for Scientific Information. In 2010, the journal was recognized by the Special Libraries Association as one of the top 100 most influential biomedical journals of the past century. FASEB is composed of 26 societies with more than 100,000 members, making it the largest coalition of biomedical research associations in the United States. Celebrating 100 Years of Advancing the Life Sciences in 2012, FASEB is rededicating its efforts to advance health and well-being by promoting progress and education in biological and biomedical sciences through service to our member societies and collaborative advocacy.

Details: Andrea Banfi, Georges von Degenfeld, Roberto Gianni-Barrera, Silvia Reginato, Milton J. Merchant, Donald M. McDonald, and Helen M. Blau. Therapeutic angiogenesis due to balanced single-vector delivery of VEGF and PDGF-BB. FASEB J. doi:10.1096/fj.11-197400 ; http://www.fasebj.org/content/early/2012/03/05/fj.11-197400.abstract

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Stanford scientists develop gene therapy approach to grow blood vessels in ischemic limbs

ScienceDaily (Mar. 6, 2012) A new research discovery by a team of Stanford and European scientists offers hope that people with atherosclerotic disease may one day be able to avoid limb amputation related to ischemia. A new research report appearing online in the FASEB Journal suggests that the delivery of genes for two molecules naturally produced by the body, called "PDGF-BB" and "VEGF" may successfully cause the body to grow new blood vessels that can save ischemic limbs.

"We hope that our findings will ultimately develop into a safe and effective therapy for the many patients, suffering from blocked arteries in the limbs, who are currently not adequately treated by surgery or drugs," said Helen M. Blau, Ph.D., a senior researcher involved in the work and Associate Editor of the FASEB Journal from the Baxter Laboratory for Stem Cell Biology at the Institute for Regenerative Medicine and Stem Cell Biology at Stanford. "This could help avoid the devastating consequences of limb amputations for both patients and their families."

To make this discovery, Blau and colleagues, including Andrea Banfi (now at Basel University), introduced the genes for PDGF-BB and VEGF into the muscles of mice, either independently or together. When high doses of VEGF alone were produced, they caused the growth of vascular tumors. When the two factors were produced in unbalanced amounts, tumor growth also occurred. When VEGF and PDGF were delivered in a fixed ratio relative to one another, however, no tumors occurred, and blood flow was restored to ischemic muscle tissue and damage repaired without any toxic effects. To achieve a "balanced" delivery of PDGF-BB and VEGF, scientists placed both genes in a single gene therapy delivery mechanism, called a "vector."

Although the report shows the feasibility of growing robust and safe new blood vessels that restore blood flow to diseased tissues, Blau points out that "there are multiple challenges to correcting peripheral vasculature disease by using proangiogenic gene therapy strategies. Two important challenges are what to deliver and how to get it to where it can have beneficial effects. Clinical success will require both delivering a gene therapy construct that encodes for effective angiogenic factors and ensuring that the sites of delivery are where the construct can have the greatest clinical benefit."

"This ingenious work, based on the latest techniques of molecular biology, tells us that it is possible to reinvigorate parts of our body that can't get enough blood to keep them going," said Gerald Weissmann, M.D., Editor-in-Chief of the FASEB Journal. "The next question is whether this approach will work in humans and exactly how to deliver the new treatment to places that need it the most."

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Gene therapy approach to grow blood vessels in ischemic limbs