换一批摘要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.
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adeno-associated virusCRISPR-Cas9developmental windowgenetic therapymouse modelsneurodevelopmental disordersnon-human primatespreclinical studies
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发布时间
2020-09-17（万方平台首次上网日期，不代表论文的发表时间）
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