rAAV-CRISPRa therapy corrects Rai1 haploinsufficiency and rescues selective disease features in Smith-Magenis syndrome mice

Haploinsufficiency in retinoic acid induced 1 (RAI1) causes Smith-Magenis syndrome (SMS), a severe neurodevelopmental disorder characterized by neurocognitive deficits and obesity. Currently, curative treatments for SMS do not exist. Here, we take a recombinant adeno-associated virus (rAAV)-clustere...

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Detalles Bibliográficos
Autores principales: Chang, Hao-Cheng, Lee, Yu-Ju, Javed, Sehrish, Haque, Minza, Chang, Ya-Ting, Lin, Yu Cheng, Oram, Cameron, Huang, Wei-Hsiang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Biochemistry and Molecular Biology 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9762195/
https://www.ncbi.nlm.nih.gov/pubmed/36410433
http://dx.doi.org/10.1016/j.jbc.2022.102728

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9762195/
https://www.ncbi.nlm.nih.gov/pubmed/36410433
http://dx.doi.org/10.1016/j.jbc.2022.102728

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