TY - JOUR
T1 - Postnatal therapeutic approaches in genetic neurodevelopmental disorders
AU - Levy, Gilad
AU - Barak, Boaz
N1 - Publisher Copyright:
© 2021 Wolters Kluwer Medknow Publications. All rights reserved.
PY - 2021/3
Y1 - 2021/3
N2 - 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.
AB - 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.
KW - CRISPR-Cas9
KW - adeno-associated virus
KW - developmental window
KW - genetic therapy
KW - mouse models
KW - neurodevelopmental disorders
KW - non-human primates
KW - preclinical studies
UR - http://www.scopus.com/inward/record.url?scp=85092630468&partnerID=8YFLogxK
U2 - 10.4103/1673-5374.293133
DO - 10.4103/1673-5374.293133
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C2 - 32985459
AN - SCOPUS:85092630468
SN - 1673-5374
VL - 16
SP - 414
EP - 422
JO - Neural Regeneration Research
JF - Neural Regeneration Research
IS - 3
ER -
