Recent advances in the pharmacology of neurological gene therapy.
The choice of vectors, transgenes, regulatory elements, delivery approaches and the capacity to transduce the appropriate target cell type all influence the effectiveness of gene therapy for neurological diseases. Furthermore, even if many strategies are sound and effective in experimental animals, issues relating to side effects of gene therapy, longevity of therapeutic transgene expression and diffusion throughout the brain can limit the clinical potential of gene therapy. During the past 12-18 months, there have been significant advances in the following areas: the capacity to target vectors to predetermined cells types; the development of gene therapy approaches for the treatment of dominant inherited and neurodegenerative diseases; the capacity to achieve systemic delivery of viral vectors to the brain; and the development of viral vectors to model neurological diseases.
Lowenstein
Pedro Ricardo
PR
Gene Therapeutics Research Institute, Cedars-Sinai Medical Center and Department of Medicine, University of California, Los Angeles, USA. lowensteinp@cshs.org
Castro
Maria Graciela
MG
D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D009422:Nervous System Diseases / Q000235:genetics / Q000628:therapy*; D010597:Pharmacogenetics / Q000379:methods / Q000639:trends*; D013997:Time Factors