Gene-replacement therapy in neurodevelopmental disorders: progress and challenges
Gene-replacement therapy in neurodevelopmental disorders: progress and challenges
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Author / Creator
Publisher
United States: American Society for Clinical Investigation
Journal title
Language
English
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Publisher
United States: American Society for Clinical Investigation
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Scope and Contents
Contents
Heterozygous loss-of-function variants in the SLC6A1 gene, encoding GAT1, which is the main GABA transporter in the brain, lead to a broad spectrum of neuropsychiatric and neurodevelopmental disorders including epilepsy, developmental delay, intellectual disability, and autism. Gene-replacement strategies involving adeno-associated viruses (AAV) re...
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Full title
Gene-replacement therapy in neurodevelopmental disorders: progress and challenges
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_11785917
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_11785917
Other Identifiers
ISSN
1558-8238,0021-9738
E-ISSN
1558-8238
DOI
10.1172/JCI188703
