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J Neurodev Disord. 2018 May 18;10(1):16. doi: 10.1186/s11689-018-9234-0.

Recent progress and considerations for AAV gene therapies targeting the central nervous system.

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Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA.
University of North Carolina at Chapel Hill, Gene Therapy Center, Chapel Hill, NC, 27599, USA.
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA.



Neurodevelopmental disorders, as a class of diseases, have been particularly difficult to treat even when the underlying cause(s), such as genetic alterations, are understood. What treatments do exist are generally not curative and instead seek to improve quality of life for affected individuals. The advent of gene therapy via gene replacement offers the potential for transformative therapies to slow or even stop disease progression for current patients and perhaps minimize or prevent the appearance of symptoms in future patients.


This review focuses on adeno-associated virus (AAV) gene therapies for diseases of the central nervous system. An overview of advances in AAV vector design for therapy is provided, along with a description of current strategies to develop AAV vectors with tailored tropism. Next, progress towards treatment of neurodegenerative diseases is presented at both the pre-clinical and clinical stages, focusing on a few select diseases to highlight broad categories of therapeutic parameters. Special considerations for more challenging cases are then discussed in addition to the immunological aspects of gene therapy.


With the promising clinical trial results that have been observed for the latest AAV gene therapies and continued pre-clinical successes, the question is no longer whether a therapy can be developed for certain neurodevelopmental disorders, but rather, how quickly.


AAV9; Adeno-associated virus; Cellular immunity; Central nervous system; Clinical trial; Gene therapy; Neutralizing antibody

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