Shortening trinucleotide repeats using highly specific endonucleases: a possible approach to gene therapy?
Abstract
•Microsatellite expansions are responsible for more than 30 neurological or developmental disorders. •Gene therapy could involve shortening expanded microsatellites using highly specific nucleases. Trinucleotide repeat expansions are involved in more than two dozen neurological and developmental disorders. Conventional therapeutic approaches aimed at regulating the expression level of affected genes, which rely on drugs, oligonucleotides, and/or...
Paper Details
Title
Shortening trinucleotide repeats using highly specific endonucleases: a possible approach to gene therapy?
Published Date
Apr 1, 2015
Journal
Volume
31
Issue
4
Pages
177 - 186
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