Gene therapy approaches to ataxias.
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, Parkinson's 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.
Lim
Filip
F
Departamento de Biología Molecular and Centro de Biología Molecular Severo Ochoa (UAM-CSIC), Universidad Autónoma de Madrid, Spain.
Diaz-Nido
Javier
J
D000818:Animals; D001259:Ataxia / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans