Gene therapy for neurodegenerative diseases.

Gene therapy for neurodegenerative diseases.
Trends Mol Med. 2015 Jun 26;
Authors: O'Connor DM, Boulis NM
Abstract
Gene therapy is, potentially, a powerful tool for treating neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, Parkinson's disease (PD) and Alzheimer's disease (AD). To date, clinical trials have failed to show any improvement in outcome beyond the placebo effect. Efforts to improve outcomes are focusing on three main areas: vector design and the identification of new vector serotypes, mode of delivery of gene therapies, and identification of new therapeutic targets. These advances are being tested both individually and together to improve efficacy. These improvements may finally make gene therapy successful for these disorders.
PMID: 26122838 [PubMed - as supplied by publisher]

Source: Trends in Molecular Medicine - June 26, 2015 Category: Molecular Biology Authors: O'Connor DM, Boulis NM Tags: Trends Mol Med Source Type: research