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
For some time, cancer scientists have considered the toxin, alpha-amanatin derived from “death cap” mushrooms, as a possible cancer treatment. However, due to its penchant for causing liver toxicity, its potential as an effective therapy has been limited.
The efficacy of gene therapy treatment for Wiskott-Aldrich syndrome has been demonstrated by researchers. Wiskott-Aldrich syndrome is a rare congenital immune and platelet deficiency which is X-linked and has an estimated prevalence of 1/250,000. It is caused by mutations in the gene encoding the WAS protein (WASp) expressed in hematopoietic cells. This disease, which primarily affects boys, causes bleeding, severe and recurrent infections, severe eczema and in some patients autoimmune reactions and the development of cancer.
Early indicators of the malaria parasite in Africa developing resistance to the most effective drug available have been confirmed, according to new research. Investigators found Plasmodium falciparum malaria parasites with a mutation to the gene Ap2mu were less sensitive to the antimalarial drug artemisinin.