Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism. There are currently no disease modifying therapies for HD; however, HTT-lowering therapies hold promise. Recombinant adeno-assoc...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Brain Sci
Main Authors: Spronck, Elisabeth A., Vallès, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2021
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7908995/
https://ncbi.nlm.nih.gov/pubmed/33498212
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/brainsci11020129
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados