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, <i>HTT</i>-lowering therapies hold promise. Recombinant aden...

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Principais autores:	Elisabeth A. Spronck, Astrid Vallès, Margit H. Lampen, Paula S. Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M. Evers, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Martin de Haan
Formato:	Artigo
Idioma:	Inglês
Publicado em:	MDPI AG 2021-01-01
coleção:	Brain Sciences
Assuntos:	AAV5 Huntington disease miRNA gene therapy
Acesso em linha:	https://www.mdpi.com/2076-3425/11/2/129
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