Gene therapy, in which a working gene is inserted into a cell to replace a faulty or absent gene, is a promising experimental technique for the prevention and treatment of disease.

Investigators at Hospital for Special Surgery have identified a new mechanism involved in the pathogenesis of inflammatory diseases such as rheumatoid arthritis. The mechanism may also shed some light on why gene therapy experiments that use adenoviruses to deliver genes to humans have run into problems. The study will appear online on March 16 in the journal Nature Immunology.

Investigators in The Research Institute at Nationwide Children’s Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

Researchers from the University of Glasgow have solved a scientific riddle that could lead to the development of more effective gene therapies.

Henry Ford Hospital is embarking on an expanded major clinical trial involving the use of gene therapy in combination with radiation therapy, to determine if the combined treatment is more effective than radiation therapy alone for patients with intermediate risk prostate cancer.

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 of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The papers are available free online.

Can a patient who agrees to participate in a safety study of a gene therapy protocol give truly informed consent and understand the risks involved when the consent forms are highly technical and the physician or institution seeking their consent has a stake in the study and its outcome?

Researchers in the Department of Medicine and Department of Neurosciences at Mount Sinai School of Medicine have discovered that chronic pain can be successfully treated with novel targeted gene therapy.

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.

Heart disease is the leading cause of death in the United States and greatly affects the quality and length of life for individuals with specific forms of muscular dystrophy.

The European Union has approved the rules that would regulate such kinds of advanced treatments as gene therapy for disease like cancer or Parkinson’s.

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.