Small molecule splicing modifiers with systemic HTT-lowering activity

Autor: Anuradha Bhattacharyya, Christopher R. Trotta, Jana Narasimhan, Kari J. Wiedinger, Wencheng Li, Kerstin A. Effenberger, Matthew G. Woll, Minakshi B. Jani, Nicole Risher, Shirley Yeh, Yaofeng Cheng, Nadiya Sydorenko, Young-Choon Moon, Gary M. Karp, Marla Weetall, Amal Dakka, Vijayalakshmi Gabbeta, Nikolai A. Naryshkin, Jason D. Graci, Thomas Tripodi, Amber Southwell, Michael Hayden, Joseph M. Colacino, Stuart W. Peltz
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Zdroj: Nature Communications, Vol 12, Iss 1, Pp 1-12 (2021)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/s41467-021-27157-z
Popis: Here the authors describe the discovery of a class of small molecule splicing modifiers which are orally bioavailable, cross the blood-brain barrier, and lower levels of huntingtin in a mouse model of Huntington’s disease (HD).
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