Nature Communications (Dec 2021)
Small molecule splicing modifiers with systemic HTT-lowering activity
- 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
Affiliations
- Anuradha Bhattacharyya
- PTC Therapeutics, Inc. 100 Corporate Court
- Christopher R. Trotta
- PTC Therapeutics, Inc. 100 Corporate Court
- Jana Narasimhan
- PTC Therapeutics, Inc. 100 Corporate Court
- Kari J. Wiedinger
- PTC Therapeutics, Inc. 100 Corporate Court
- Wencheng Li
- PTC Therapeutics, Inc. 100 Corporate Court
- Kerstin A. Effenberger
- PTC Therapeutics, Inc. 100 Corporate Court
- Matthew G. Woll
- PTC Therapeutics, Inc. 100 Corporate Court
- Minakshi B. Jani
- PTC Therapeutics, Inc. 100 Corporate Court
- Nicole Risher
- PTC Therapeutics, Inc. 100 Corporate Court
- Shirley Yeh
- PTC Therapeutics, Inc. 100 Corporate Court
- Yaofeng Cheng
- PTC Therapeutics, Inc. 100 Corporate Court
- Nadiya Sydorenko
- PTC Therapeutics, Inc. 100 Corporate Court
- Young-Choon Moon
- PTC Therapeutics, Inc. 100 Corporate Court
- Gary M. Karp
- PTC Therapeutics, Inc. 100 Corporate Court
- Marla Weetall
- PTC Therapeutics, Inc. 100 Corporate Court
- Amal Dakka
- PTC Therapeutics, Inc. 100 Corporate Court
- Vijayalakshmi Gabbeta
- PTC Therapeutics, Inc. 100 Corporate Court
- Nikolai A. Naryshkin
- PTC Therapeutics, Inc. 100 Corporate Court
- Jason D. Graci
- PTC Therapeutics, Inc. 100 Corporate Court
- Thomas Tripodi
- PTC Therapeutics, Inc. 100 Corporate Court
- Amber Southwell
- Burnett School of Biomedical Sciences, College of Medicine, University of Central Florida
- Michael Hayden
- Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, University of British Columbia
- Joseph M. Colacino
- PTC Therapeutics, Inc. 100 Corporate Court
- Stuart W. Peltz
- PTC Therapeutics, Inc. 100 Corporate Court
- DOI
- https://doi.org/10.1038/s41467-021-27157-z
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 12
Abstract
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).
