Gene Therapy Review
Welcome to our gene therapy portal
The Gene Therapy Review is the web resource for patients and professionals interested in cell and gene therapy. Founded in 2008, the aim of the site is to be an information resource for basic and clinical research in the fields of gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to this arena. We welcome contributions from researchers and clinicians alike with a view in increase awareness in this vastly growing sector.
Gene Therapy in the News
Researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). This extremely rare genetic disorder causes children to gradually lose the ability to balance themselves, move their muscles and to feel certain sensations. Most children born with GAN do not survive beyond their early 20s because of progressive impairment of their ability to breath.
The development of targeted therapies has significantly improved the survival of melanoma patients over the last decade; however, patients often relapse because many therapies do not kill all of the tumor cells, and the remaining cells adapt to treatment and become resistant. Researchers have discovered a novel mechanism that can lead melanoma cells to develop resistance to drugs that target the protein BRAF.
Gene therapy to reduce production of a brain protein prevented development of Parkinson's disease in an animal study, researchers report. The findings could lead to new understanding of how genetic and environmental factors converge to cause the disease, and the development of effective treatments to prevent disease progression.