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American Society for Experimental Neurotherapeutics Abstracts
August 2001

Gene Transfer to the Nervous System: Status and Promise for Therapy

Arch Neurol. 2001;58(8):1316. doi:

The rapid development of many platforms for gene transfer to the central nervous system (CNS) has made it possible to contemplate therapeutic applications. However, as issues remain unanswered, it is clear that a systematic, deliberate, and rigorous path must be taken to achieve this goal. This lecture will review a number of considerations relevant to the implementation of gene transfer for CNS disease. These topics include methods of CNS gene delivery, potential clinical targets and their special considerations, safety issues, and extant impediments to successful clinical translation. (Supported by NIH grants R01NS36420, R01ES09391, R01MH57047, and P30AG18254.)