Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.

Over-expressing a gene that lets brain cells communicate just a fraction of a second longer makes a smarter rat, report researchers from the Medical College of Georgia and East China Normal University.

Scientists from the Universities of Michigan and Minnesota show in a research report published online in the FASEB Journal (http://www.fasebj.org) that gene therapy may be used to improve an ailing heart's ability to contract properly. In addition to showing gene therapy's potential for reversing the course of heart failure, it also offers a tantalizing glimpse of a day when "closed heart surgery" via gene therapy is as commonly prescribed as today's cocktail of drugs.

An international team of scientists and clinicians from the United States and Saudi Arabia are working to develop gene therapy for treating a rare, hereditary retinal disease. The therapy has been shown to restore lost vision in animal models of retinitis pigmentosa (RP). Their work is being funded in part by a $1.5 million grant from the Prince Salman Center for Disability Research in Saudi Arabia, where the recessive gene mutation that leads to the eye disease RP has been found in children from several families.

University of Florida researchers have come up with a new gene therapy method to disrupt cancer growth by using a synthetic protein to induce blood clotting that cuts off a tumor's blood and nutrient supply.

With obesity reaching epidemic levels, researchers at the Ohio State University Medical Center are studying a potentially long-term treatment that involves injecting a gene directly into one of the critical feeding and weight control centers of the brain.

A new UCLA AIDS Institute study has found that gene therapy can be developed as a safe and active technique to combat HIV.

Long-term gene therapy resulted in improved cardiac function and reversed deterioration of the heart in rats with heart failure, according to a recent study conducted by researchers at Thomas Jefferson University's Center for Translational Medicine. The study was published online in Circulation.

Scientists at the University of Michigan have shown that gene therapy can be used to successfully stop the development of periodontal disease, the leading cause of tooth loss in adults.

The apparent success of a case in which German doctors cured a man of AIDS using a bone marrow transplant comes as no surprise to Gerhard Bauer, a UC Davis stem cell researcher. Bauer has been working for more than 10 years on a similar cure for AIDS based on replacing the devastated immune system of an HIV-infected patient with stem cells that have been engineered to resist human immunodeficiency syndrome.

Using a harmless virus to insert a corrective gene into mouse blood cells, scientists at St. Jude Children's Research Hospital have alleviated sickle cell disease pathology. In their studies, the researchers found that the treated mice showed essentially no difference from normal mice. Although the scientists caution that applying the gene therapy to humans presents significant technical obstacles, they believe that the new therapy will become an important treatment for the disease.

Massachusetts General Hospital (MGH) researchers have used gene therapy to restore useful vision to mice with degeneration of the light-sensing retinal rods and cones, a common cause of human blindness. Their report, appearing in the Oct. 14 Proceedings of the National Academy of Sciences, describes the effects of broadly expressing a light-sensitive protein in other neuronal cells found throughout the retina.

The gene transfer vector (left) is injected into the skin in the area of pain (red line, right). From the skin, the vector is carried into sensory nerves and releases the inhibitory neurotransmitter locally in the spinal cord (red oval). Credit: University of Michigan This week, University of Michigan scientists will begin a phase 1 clinical trial for the treatment of cancer-related pain, using a novel gene transfer vector injected into the skin to deliver a pain-relieving gene to the nervous system.

A new approach to treating vision loss caused by Type 1 Usher syndrome (USH1), the most common condition affecting both sight and hearing, will be unveiled by a scientist at the annual conference of the European Society of Human Genetics tomorrow (Tuesday 3 June). Ms Annie Rebibo Sabbah, from the Genetics Department of the Rappaport Faculty of Medicine, Technion, Haifa, Israel, will tell the conference that preliminary results using a class of drugs called aminoglycosides, commonly used as antibiotics, had had promising effects in vitro and in cell culture.

Research led by investigators at Memorial Sloan-Kettering Cancer Center (MSKCC) has shown that therapeutic cloning, also known as somatic-cell nuclear transfer (SCNT), can be used to treat Parkinson’s disease in mice. The study’s results are published in the March 23 online edition of the journal Nature Medicine.

By injecting a customized "genetic patch" into early stage fish embryos, researchers at Washington University School of Medicine in St. Louis were able to correct a genetic mutation so the embryos developed normally.

A gene therapy treatment that restores a missing liver enzyme in test animals could provide a cure for a rare metabolic disorder in humans, according to Duke University Medical Center researchers.

A research team at the Moores Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were treated with a gene therapy protocol began making antibodies that reacted against their own leukemia cells. The study will be published on line the week of February 11-15 in the online edition of the Proceedings of the National Academy of Science.

RNA interference (RNAi) represents an innovative new strategy for using small RNA molecules to silence specific genes associated with disease processes, and a series of review articles describing the state-of-the-art and potential therapeutic applications of RNAi and microRNAs will begin with two review papers in the January 2008 issue (Volume 19, Number 1) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The papers are available free online.

Researchers at Johns Hopkins and Ohio State University have found that the number of copies of a particular gene can affect the severity of colon cancer in a mouse model. Publishing in the Jan. 3 issue of Nature, the research team describes how trisomy 21, or Down syndrome in humans, can repress tumor growth.

Mice with a human sickle-cell anemia disease trait have been treated successfully in a process that begins by directly reprogramming their own cells to an embryonic-stem-cell-like state, without the use of eggs. This is the first proof-of-principle of therapeutic application in mice of directly reprogrammed “induced pluripotent stem” (IPS) cells, which recently have been derived in mice as well as humans.

Three decades have passed since gene therapy pioneer William W. Hauswirth, Ph.D., and his colleagues at the University of Florida began work on a virus that could safely deliver corrective genes into living animals.

Gene therapy administered intravenously could be an effective agent to protect vital organs and tissues from the effects of ionizing radiation in the event of large-scale exposure from a radiological or nuclear bomb, according to an animal study presented today by University of Pittsburgh researchers at the 49th annual meeting of the American Society for Therapeutic Radiology and Oncology (ASTRO) in Los Angeles.

Researchers at the Board of Governors Gene Therapeutics Research Institute at Cedars-Sinai Medical Center have shown for the first time that it is possible to sustain therapeutic gene expression in the central nervous system for up to a year, even in the presence of an anti-viral immune response mechanism that is normally present in humans.

By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brain—after one injection of gene therapy. If these results in animals can be realized in people, researchers may have a potential method for gene therapy to treat a host of rare but devastating congenital human neurological disorders, such as Tay-Sachs disease.

To move a gene from point A to point B, scientists and gene therapists have two proven options: a virus, which can effectively ferry genes of interest into cells, and a plasmid, an engineered loop of DNA that can do the same thing, albeit usually only on a short-term basis.

Researchers from MIT, Alnylam Pharmaceuticals and other institutions have demonstrated the safety of a promising type of genetic therapy that could lead to treatments for a wide range of diseases such as cancer.

Neurologix, Inc. (OTC Bulletin Board: NRGX) today announced the publication in the June 23 issue of the journal The Lancet of positive results from the first ever gene therapy trial for Parkinson’s disease and the first report of direct gene transfer into a patient’s own brain cells for any adult neurodegenerative disease.

Rats with erectile dysfunction, or ED, that were injected with a gene therapy vector containing either of two nerve growth factors were able to regain normal function after four weeks, according to a study conducted by University of Pittsburgh School of Medicine researchers. These findings are being presented at the 10th annual meeting of the American Society of Gene Therapy, which is convening May 30 to June 3 at the Washington State Convention & Trade Center, Seattle.

Early-stage research has found that a new gene therapy can nearly eliminate arthritis pain, and significantly reduce long-term damage to the affected joints, according to a study published today in the journal Arthritis and Rheumatism. While the study was done in mice, they are the first genetically engineered to develop osteoarthritis like humans, with the same genetic predisposition that makes some more likely to develop the disease, the authors said. If all goes well with a follow-up study currently underway, researchers will apply to the U.S. Food and Drug Administration for permission to begin human trials next year.