gene-cell-therapy

Gene Therapy is the transfer of genetic material (gene transfer) to dysfunctional cells to correct a deficiency in the DNA or genome of a patient. Cell Therapy, such as stem cell therapy, can also be used to achieve these goals. These approaches can be applied to genetic disorders as well as diseases acquired over the lifetime of an individual, such as cancer or infection, to confer a specific property to the cell allowing it to combat the disease.

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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.

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© 2012 Gene Therapy Review. All Rights Reserved. ISSN (print) 1792-0094, ISSN (online) 1792-0108