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.

The first clinical trial to test a revolutionary treatment for blindness in children has been announced by researchers at UCL (University College London). The trial, funded by the Department of Health, is the first of its kind and could have a significant impact on future treatments for eye disease.

A cancer-suppressing gene has been successfully delivered into the tumors of stage 4 lung cancer patients via an intravenously administered lipid nanoparticle in a phase I clinical trial at The University of Texas M. D. Anderson Cancer Center. The gene, FUS1, also was found to be active in the metastatic non-small cell lung cancer tumors.

Gene therapy - the idea of using genetic instructions rather than drugs to treat disease - has tickled scientists' imaginations for decades, but is not yet a viable therapeutic method. One sizeable hurdle is getting the right genes into the right place at the right time.

University of Florida researchers have used an experimental therapy in mice to shut down a gene that plays a crucial role in a leading cause of inherited blindness.

Since the late 1980s, gene therapy, more than virtually any other type of therapy, has given rise not only to high expectations of treatment success but also great concerns regarding health risks. Since the Deutsche Forschungsgemeinschaft (German Research Foundation, DFG) issued its first memorandum in 1995, this field of research has developed enormously.

Researchers at the University of Pennsylvania’s School of Medicine have found by targeting the function of a single gene that it is possible to inhibit bone decay while simultaneously stimulating bone formation. This concept may lead to drug treatments for osteoporosis and other bone diseases. Senior author Yongwon Choi, PhD, professor of Pathology and Laboratory Medicine at the University of Pennsylvania and colleagues report their findings in the December issue of Nature Medicine.

The first human study using gene transfer to treat erectile dysfunction (ED) shows promising results and suggests the potential for using the technology to treat overactive bladder, irritable bowel syndrome and asthma, according to the researchers.

An experimental gene therapy to combat alpha-1 antitrypsin deficiency, a common hereditary disorder that causes lung and liver disease, has caused no harmful effects in patients and shows signs of being effective, University of Florida researchers say.

therapy administered intravenously could be used as an 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 48th Annual Meeting of the American Society for Therapeutic Radiology and Oncology (ASTRO) in Philadelphia.

For the first time, researchers have inhibited the development of epilepsy after a brain insult in animals. By using gene therapy to modify signaling pathways in the brain, neurology researchers found that they could significantly reduce the development of epileptic seizures in rats.

A protein with the ironic name "Srcasm" can counteract the effects of tumor-promoting molecules in skin cells, according to new research by investigators at the University of Pennsylvania School of Medicine. Using animal models, the researchers discovered that Srcasm acts like a brake in epithelial cells, preventing uncontrolled cell growth caused by a family of proteins called Src kinases. This finding, published online in the Journal of Biological Chemistry, suggests a target for future gene therapy to treat skin, head, neck, colon, and breast cancers.

Scientists at Case Western Reserve University School of Medicine and Albany Medical College, together with Rainforest Nutritionals, Inc. have demonstrated in a mini symposium held today at the 14th International Conference of the Inflammation Research Association (IRA) that a blend of natural botanical products called Reparagen have turned on the master repair gene, IGF-1, which is responsible for growing human cartilage and restores joint function while blocking joint destruction associated with inflammation. The IRA is the preeminent meeting for inflammation investigators.

Transfer of a gene that produces a mutant form of good cholesterol provides significantly better anti-plaque and anti-inflammation benefits than therapy using the "normal" HDL gene, according to a mouse study conducted by cardiology researchers at Cedars-Sinai Medical Center and reported in the Oct. 3 issue of the Journal of the American College of Cardiology.

A jumping gene first identified in a cabbage-eating moth may one day provide a safer, target-specific alternative to viruses for gene therapy, researchers say.