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

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

Abstract Background 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 affe...

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Bibliographic Details
Main Authors: Erik Allen Lykken, Charles Shyng, Reginald James Edwards, Alejandra Rozenberg, Steven James Gray
Format: Article
Language:English
Published: BMC 2018-05-01
Series:Journal of Neurodevelopmental Disorders
Subjects:
Central nervous system
Adeno-associated virus
AAV9
Gene therapy
Clinical trial
Neutralizing antibody
Online Access:http://link.springer.com/article/10.1186/s11689-018-9234-0

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http://link.springer.com/article/10.1186/s11689-018-9234-0

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