Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates

Huntington's disease is a fatal neurodegenerative disorder caused by an inherited mutation in the huntingtin (HTT) gene comprising an expanded cytosine-adenine-guanine (CAG) trinucleotide repeat sequence that results in a pathogenic huntingtin protein. Adeno-associated viral (AAV) gene therapy...

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Detalles Bibliográficos
Autores principales: Wang, Wei, Zhou, Pengcheng, Wang, Xin, Chen, Fen, Christensen, Emily, Thompson, Jeffrey, Ren, Xiaoqin, Kells, Adrian, Stanek, Lisa, Carter, Todd, Hou, Jay, Sah, Dinah W.Y.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10112875/
https://www.ncbi.nlm.nih.gov/pubmed/34806402
http://dx.doi.org/10.1089/hum.2021.221

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10112875/
https://www.ncbi.nlm.nih.gov/pubmed/34806402
http://dx.doi.org/10.1089/hum.2021.221

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