Postnatal therapeutic approaches in genetic neurodevelopmental disorders  

作　　者：Gilad Levy Boaz Barak 

机构地区：[1]The Sagol School of Neuroscience,Tel Aviv University,Tel Aviv,Israel [2]The School of Psychological Sciences,Faculty of Social Sciences,Tel Aviv University,Tel Aviv,Israel

出　　处：《Neural Regeneration Research》2021年第3期414-422,共9页中国神经再生研究（英文版）

基　　金：supported by grants from Fritz Thyssen Stiftung,Brain Boost Innovation Center by Sagol School of Neuroscience at TAU,and SPARK Tel Aviv;supported by the Eshkol Fellowship from The Ministry of Science and Technology;the recipient of The Alon Fellowship for outstanding young researchers awarded by the Israeli Council for Higher Education。

摘　　要：Genetic neurodevelopmental disorders are characterized by abnormal neurophysiological and behavioral phenotypes,affecting individuals worldwide.While the subject has been heavily researched,current treatment options relate mostly to alleviating symptoms,rather than targeting the altered genome itself.In this review,we address the neurogenetic basis of neurodevelopmental disorders,genetic tools that are enabling precision research of these disorders in animal models,and postnatal gene-therapy approaches for neurodevelopmental disorders derived from preclinical studies in the laboratory.

关 键 词：adeno-associated virus CRISPR-Cas9 developmental window genetic therapy mouse models neurodevelopmental disorders non-human primates preclinical studies 

分 类 号：R741.05[医药卫生—神经病学与精神病学]