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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Brain Sci
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: MDPI 2021
Aiheet:
Article
Linkit: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
Tagit: Lisää tagi
Ei tageja, Lisää ensimmäinen tagi!

Samankaltaisia teoksia