Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s Disease-Reference-Cited by-同舟云学术

Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s Disease

Published:2023-09-15 Issue:18 Volume:66 Page:13205-13246
ISSN:0022-2623
Container-title:Journal of Medicinal Chemistry
language:en
Short-container-title:J. Med. Chem.

Author:

Liu Longbin¹^ORCID,Malagu Karine²,Haughan Alan F.²,Khetarpal Vinod¹,Stott Andrew J.²,Esmieu William²,Vater Huw D.²^ORCID,Webster Stephen J.²,Van de Poël Amanda J.²,Clissold Cole²,Cosgrove Brett²,Sutton Benjamin²,Spencer Jonathan A.²,Breccia Perla²^ORCID,Gancia Emanuela²,Bonomo Silvia²,Ladduwahetty Tammy²,Lazari Ovadia²,Patel Hiral²,Atton Helen C.²,Clifton Steve²,Mota Daniel M.²,Magnani Dario²,O’Neill Amy²,Stebbeds Marta²,Macabuag Natsuko²,Todd Daniel²,Herva Maria E.²,Mitchell Philip²,Visser Mijke³,Compte Sancerni Sara³^ORCID,Grand Moursel Laure³,da Silva Marta³,Kritikou Eva³,Heikkinen Taneli T.⁴,Bolkvadze Tamuna⁴,Fodale Valentina⁵,Spadafora Debora⁵,Daldin Manuel⁵,Bresciani Alberto⁵,Mangette John E.⁶,Doherty Elizabeth M.¹,Lee Matthew R.¹,Herbst Todd¹,Monteagudo Edith¹,Macdonald Douglas¹,Plotnikov Nikolay V.¹^ORCID,Chambers Mark²,McAllister George¹,Muňoz-Sanjuan Ignacio¹,Dominguez Celia¹

Affiliation:

1. CHDI Management/CHDI Foundation, 6080 Center Drive, Los Angeles, California 90045, United States

2. Discovery from Charles River, Charles River, Chesterford Research Park, Saffron Walden CB10 1XL, U.K.

3. Charles River, Darwinweg 24, 2333 CR Leiden, The Netherlands

4. Charles River, Microkatu 1, Kuopio 70210 Finland

5. IRBM S.p.A., Pomezia, Roma 00071, Italy

6. Curia Global, Inc., Buffalo, New York 14203, United States

Funder

CHDI Foundation

Publisher

American Chemical Society (ACS)

Subject