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A gene therapy that uses infusions of patients’ own T cells genetically engineered to attack their tumors enjoyed its first successful and sustained demonstration of clinical-trial success in nine of 12 leukemia patients—two of whom have been in remission for more than two years. The therapy was pioneered by the University of Pennsylvania’s Perelman School of Medicine, whose researchers will present latest results from the study today at the American Society of Hematology (ASH)'s Annual Meeting and Exposition in Atlanta.According to Penn Perelman, the results pave the way for a potential paradigm shift in the treatment of these types of...

Mitochondrial transfer could reduce the risk of childhood disease. Researchers say that technology to shuffle genetic material between unfertilized eggs is ready to make healthy babies. The technique could allow parents to minimize the risk of a range of diseases related to defects in the energy-producing cell organelles known as mitochondria. Mitochondrial defects affect an estimated 1 in 4,000 children, and can cause rare and often fatal diseases such as carnitine deficiency, which prevents the body from using fats for energy. They are also implicated in a wide range of more common diseases affecting children and adults, such as multiple...

A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum). "This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. In...

Enlarge Image Gene fix. Red cells in this slice of mouse liver are making a human protein called A1At. Credit: K. YUSA ET AL., NATURE (ADVANCED ONLINE EDITION) ©2011 MACMILLAN PUBLISHERS LTD. Researchers have taken a step toward showing how stem cells might one day be used to help patients born with a deadly liver disease. The researchers corrected a DNA spelling error in patient skin cells that had been converted into so-called induced pluripotent stem (iPS) cells, then coaxed the cells to form liver cells that seemed to function normally in mice. The approach is still a long...

Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.It means patients with a genetic disease could, one day, be treated with their own cells. A study in Nature corrected a mutation in stem cells made from a patient with a liver disease. Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed. At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would...

A gene silencing approach can save monkeys from high doses of the most lethal strain of Ebola virus in what researchers call the most viable route yet to treating the deadly and frightening infection. They used small interfering RNAs or siRNAs, a new technology being developed by a number of companies, to hold the virus at bay for a week until the immune system could take over. Tests in four rhesus monkeys showed that seven daily injections cured 100 per cent of them. U.S. government researchers and a small Canadian biotech company, Tekmira Pharmaceuticals, worked together to develop the new...

Functionalised gold nanoparticles make a more efficient and versatile delivery system for use in gene therapy and cancer treatment, claim Korean scientists. Gold nanoparticles are non-toxic and easily made and so provide an effective way to deliver therapeutic antisense DNA to disease causing genes. Antisense DNA that is covalently attached to the nanoparticles will bind to messenger RNA of a disease causing gene, preventing translation and therefore inactivating it. But previous systems could deliver only antisense DNA that was covalently cross-linked to gold nanoparticles, which needed to be individually synthesized for each gene of interest. Now, Kangseok Lee and Min Su Han at...

Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness. Achromatopsia is a rare autosomal recessive disorder with an estimated prevalence in human beings of about 1 in 30,000 to 50,000. It primarily affects the function of the cone photoreceptors in the retina and serves as a representative model for other more common inherited retinal disorders affecting cones. Cone function is essential for color vision, central visual acuity and most daily visual activities, which underlines...

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection. These new findings suggest that patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the...

(BRONX, NY) — A team led by researchers at Albert Einstein College of Medicine of Yeshiva University has found a clear link between living to 100 and inheriting a hyperactive version of an enzyme that rebuilds telomeres — the tip ends of chromosomes. The findings appear in the latest issue of the Proceedings of the National Academy of Sciences. Telomeres play crucial roles in aging, cancer and other biological processes. Their importance was recognized last month, when three scientists were awarded the 2009 Nobel Prize in Physiology and Medicine for determining the structure of telomeres and discovering how they protect...

A treatment based on HIV finds first success in humans.Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells. The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now. Together with his colleagues, paediatric neurologist Patrick Aubourg at INSERM — France's main biomedical research agency — and at the Saint-Vincent de Paul Hospital in Paris, developed the system to treat X-linked adrenoleukodystrophy (ALD), a neurodegenerative disease that affects young...

Introducing three genes corrects motor defects in monkeys.A potential gene therapy for Parkinson's disease can correct motor deficits in monkeys without causing the jerky, involuntary movements that often accompany long-term treatments for the disease. The approach is undergoing preliminary testing in a handful of human patients, who have all shown promising signs of improvement.At present, the most common remedy for Parkinson's disease involves replacing dopamine — the neurotransmitter that is depleted in patients with the disease — by administering the dopamine precursor levodopa, or L-DOPA. Most patients initially regain near-normal motor control, but after several years on L-DOPA the majority...

Dalton, a squirrel monkey treated with gene therapy, enjoys his new colour sense.Neitz Laboratory Researchers have used gene therapy to restore colour vision in two adult monkeys that have been unable to distinguish between red and green hues since birth — raising the hope of curing colour blindness and other visual disorders in humans."This is a truly amazing study," says András Komáromy, a vision researcher and veterinary ophthalmologist at the University of Pennsylvania in Philadelphia, who was not involved in the research. "If we can target gene expression specifically to cones [in humans] then this has a tremendous implication."About 1...

Enlarge ImageOn track. Colonies of genetically corrected cells taken from Fanconi anemia patients show red and yellow, markers associated with pluripotency. Credit: Juan Carlos Izpisúa Belmonte Two papers published this week appear to bring closer the day when embryonic-like stem cells can be used to treat human diseases. One study describes what scientists say is the safest method yet to produce these cells. The other reports success in using the cells to begin correcting a rare genetic disorder known as Fanconi anemia. Induced pluripotent stem (iPS) cells were first reported in 2006 by Shinya Yamanaka, a researcher at Kyoto...

Enlarge ImageDesigner genes. Monkeys given a virus that holds the DNA for lab-made antibodies dodged SIV.Credit: Malcolm Linton Researchers are reporting that a new antiviral strategy powerfully protects monkeys from SIV, the simian cousin of HIV. The approach combines elements of vaccines and gene therapy, and experts say the development could eventually lead to a vaccinelike weapon against AIDS--a goal that has thus far proved elusive. Vaccines work by priming the "adaptive" immune system to recognize and attack a specific invader. But despite 2 decades of research, several potential AIDS vaccines have failed to teach the immune system to...

A radical technique for treating diabetes could recruit cells in the gut to make insulin SAN DIEGO — If your pancreas fails you, go with your gut. Inserting a gene into gut cells in mice enabled those cells to take over the pancreas’s job, producing insulin after meals, according to unpublished research announced June 18 in San Diego at the Biotechnology Industry Organization International Convention. The work may offer a novel way to treat diabetes. "This is the first time that we've engineered a tissue that is not the pancreas to manufacture insulin" in animals, says researcher Anthony Cheung, a...

Enlarge ImageSeeing the light.Steven Howarth, 18, a patient in a U.K. gene-therapy trial for blindness, says he is now more comfortable walking home at dusk.Credit: BBC In what eye researchers are hailing as a major advance, gene therapy has partially restored the sight of four young adults born with severe blindness. In two small studies, the patients' ability to sense light improved, and two can now read several lines of an eye chart. All are still legally blind, but the same treatment could potentially prevent this type of blindness in babies. The four patients have a disease called Leber...

Closure? The death of Jolee Mohr, shown here with her family, was unrelated to the gene-therapy treatment she received, the study's sponsor says.Credit: Mohr Family/AP Images Federal regulators have given a green light to a gene-therapy arthritis trial that was halted last summer after a patient died. New tests indicate that the therapy played no role in the death. The decision comes as a relief to gene-therapy researchers who had worried about a potential new setback for their field. The trial conducted by Targeted Genetics Corp. was shut down after the 24 July death of 36-year-old Jolee Mohr of Taylorville,...

Heart attacks are the leading cause of death in both men and women, with over 450,000 deaths in the United States each year. Improving the treatments available to patients who have survived a heart attack is therefore imperative. Researchers from the National Center for Regenerative Medicine (NCRM) have identified two innovative and distinctly different methods to help treat patients recovering from a heart attack.  The first study showed that cell-based gene therapy to regenerate damaged cardiac muscle tissue and improve mechanical cardiac function was feasible using skeletal muscle stem cells modified to express a stem cell honing signal. The second...

A new discovery in stem cell research may mean big things for cancer patients in the future. Gary Van Zant, Ph.D., and a research team at the University of Kentucky published their findings today in Nature Genetics, an international scientific journal. The researchers genetically mapped a stem cell gene and its protein product, Laxetin, and building on that effort, carried the investigation all the way through to the identification of the gene itself. This is the first time such a complete study on a stem cell gene has been carried out. This particular gene is important because it helps regulate...