Cargando…
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...
| Autores principales: | , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer International Publishing
2022
|
| Materias: | |
| 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 |