Prospects for gene therapy in inherited neurodegenerative diseases

The use of gene therapy to correct or replace deficient genes has been a long-standing aspiration.

Recent findings from basic and applied research suggest that at last it may be possible to translate experimental procedures into effective patient therapies for genetic diseases. Therapies for neurodegenerative diseases potentially include, as their targets, both monogenic conditions (e.g. lysosomal storage disorders) and more genetically complex diseases (such as Alzheimer's and Parkinson's disorders).

The use of gene therapy to target the central nervous system presents specific technical and biological challenges. These may be overcome by using novel gene vector delivery strategies. Current research should illuminate the temporal window required to achieve a successful therapy. As greater knowledge is accumulated about gene therapy, correlations will be made between the level of gene expression from the therapeutic vector, the extent of correction after treatment, and the stage of disease progression when therapy is initiated.