Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic

Huntington disease (HD) is a fatal progressive neurodegenerative disorder caused by an inherited mutation in the huntingtin (HTT) gene, which encodes mutant HTT protein. Though HD remains incurable, various preclinical studies have reported a favorable response to HTT suppression, emphasizing HTT lo...

Descripción completa

Detalles Bibliográficos
Autores principales: Rook, Morgan E., Southwell, Amber L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Leading Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8899000/
https://www.ncbi.nlm.nih.gov/pubmed/35254632
http://dx.doi.org/10.1007/s40259-022-00519-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8899000/
https://www.ncbi.nlm.nih.gov/pubmed/35254632
http://dx.doi.org/10.1007/s40259-022-00519-9

Ejemplares similares