CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice

Fragile X syndrome is a neurodevelopmental disorder caused by the absence of the mRNA-binding protein fragile X messenger ribonucleoprotein (FMRP). Because FMRP is a highly pleiotropic protein controlling the expression of hundreds of genes, viral vector–mediated gene replacement therapy is viewed a...

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Detalles Bibliográficos
Autores principales: Wong, Hayes, Hooper, Alexander W.M., Kang, Hye Ri, Lee, Shiron J., Zhao, Jiayi, Sadhu, Chanchal, Rawat, Satinder, Gray, Steven J., Hampson, David R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393223/
https://www.ncbi.nlm.nih.gov/pubmed/37288657
http://dx.doi.org/10.1172/jci.insight.169650

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393223/
https://www.ncbi.nlm.nih.gov/pubmed/37288657
http://dx.doi.org/10.1172/jci.insight.169650

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