Gene Therapy Approaches to Ataxias

Title: Gene Therapy Approaches to Ataxias

Volume: 9 Issue: 1

Author(s): Filip Lim and Javier Diaz-Nido

Affiliation:

Keywords: Viral vectors, gene transfer, gene delivery, herpesvirus, Friedreich's ataxia, neuromuscular disorders, neurodegeneratio

Abstract: Gene therapy has been a clinical possibility since 1989 and the steadily increasing number of clinical trials now includes strategies targeting neurodegenerative conditions such as lysosomal storage disease, multiple sclerosis, Alzheimer s and, Parkinsons disease. In spite of lack of knowledge of the molecular causes of these diseases, results so far in these trials have been promising. Thus there is gaining confidence in the potential to develop effective treatments based on gene transfer for neurological diseases in the near future. Furthermore, the accelerating progress in knowledge of the molecular pathologies of neurogenetic disorders, including rare diseases such as the ataxias, makes them even more amenable to gene therapy. Here we review recent preclinical studies relevant to gene therapy of ataxias and discuss developments needed to bring these strategies into the clinic.

Cite this article as:

Lim Filip and Diaz-Nido Javier, Gene Therapy Approaches to Ataxias, Current Gene Therapy 2009; 9 (1) . https://dx.doi.org/10.2174/156652309787354649