CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice
Author:
Funder
Taysha Gene Therapies Inc.
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://insight.jci.org/articles/view/169650/files/pdf
Reference56 articles.
1. Fragile X mental retardation protein replacement restores hippocampal synaptic function in a mouse model of fragile X syndrome
2. FMRP Expression Levels in Mouse Central Nervous System Neurons Determine Behavioral Phenotype
3. Reduced Phenotypic Severity Following Adeno-Associated Virus-Mediated Fmr1 Gene Delivery in Fragile X Mice
4. Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity
5. Gene therapy using human FMRP isoforms driven by the human FMR1 promoter rescues fragile X syndrome mouse deficits
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