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Neuropharmacology. 2013 May;68:136-42. doi: 10.1016/j.neuropharm.2012.06.024. Epub 2012 Jun 27.

Gene therapy and neurodevelopmental disorders.

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Gene Therapy Center, University of North Carolina at Chapel Hill, 7109 Thurston Bowles, 104 Manning Drive, Chapel Hill, NC 27599-7352, USA.


With a number of recent clinical successes, gene therapy is quickly becoming a realistic treatment option for neurological disorders. Advancements in global central nervous system (CNS) gene delivery, in particular, have accelerated to the point that treatments for neurological disorders such as lysosomal storage diseases seem within reach. Other neurodevelopmental disorders, such as Rett Syndrome, Fragile X, and autism still face significant obstacles to overcome before a viable human gene therapy can be considered. This review focuses on the most common CNS gene delivery vehicle, adeno-associated virus (AAV), and the current state of AAV vector design and delivery for CNS gene therapy. Relevant examples of gene therapy studies for neurodevelopmental disorders, as well as outstanding challenges, are discussed. This article is part of the Special Issue entitled 'Neurodevelopmental Disorders'.

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