A pathogenic proteolysis–resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function

Huntington’s disease (HD) is a late-onset neurological disorder for which therapeutics are not available. Its key pathological mechanism involves the proteolysis of polyglutamine-expanded (polyQ-expanded) mutant huntingtin (mHTT), which generates N-terminal fragments containing polyQ, a key contribu...

Descripción completa

Detalles Bibliográficos
Autores principales: Kim, Hyeongju, Lenoir, Sophie, Helfricht, Angela, Jung, Taeyang, Karneva, Zhana K., Lee, Yejin, Beumer, Wouter, van der Horst, Geert B., Anthonijsz, Herma, Buil, Levi C.M., van der Ham, Frits, Platenburg, Gerard J., Purhonen, Pasi, Hebert, Hans, Humbert, Sandrine, Saudou, Frédéric, Klein, Pontus, Song, Ji-Joon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9536263/
https://www.ncbi.nlm.nih.gov/pubmed/35943803
http://dx.doi.org/10.1172/jci.insight.154108

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9536263/
https://www.ncbi.nlm.nih.gov/pubmed/35943803
http://dx.doi.org/10.1172/jci.insight.154108

Ejemplares similares