Journal
CURRENT GENE THERAPY
Volume 9, Issue 1, Pages 1-8Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/156652309787354649
Keywords
Viral vectors; gene transfer; gene delivery; herpesvirus; Friedreich's ataxia; neuromuscular disorders; neurodegeneratio
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Funding
- Spanish Ministry of Education and Science [SAF2006-12782C03-02]
- Autonomous Government of Madrid (Comunidad Autonoma de Madrid, CAM) through the NEURODE GMODELS [S-SAL-0202- 2006]
- Fundacion Alicia Koplowitz
- Association Francaise de l'Ataxie de Friedreich
- Friedreich's Ataxia Research Alliance
- Instituto de Salud Carlos III (ISCIII)
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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.
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