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

Journal

Journal of medicinal chemistry
ISSN: 1520-4804
Titre abrégé: J Med Chem
Pays: United States
ID NLM: 9716531

Informations de publication

Date de publication:
28 Sep 2023
Historique:
pubmed: 15 9 2023
medline: 15 9 2023
entrez: 15 9 2023
Statut: ppublish

Résumé

Huntington's disease (HD) is caused by an expanded CAG trinucleotide repeat in exon 1 of the huntingtin (

Identifiants

DOI: 10.1021/acs.jmedchem.3c01173 PMID: 37712656
pubmed: 37712656
doi: 10.1021/acs.jmedchem.3c01173
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

13205-13246

Auteurs

Longbin Liu (L)

CHDI Management/CHDI Foundation, 6080 Center Drive, Los Angeles, California 90045, United States.
ORCID: 0000-0003-3995-0196

Karine Malagu (K)

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

Alan F Haughan (AF)

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

Vinod Khetarpal (V)

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

Andrew J Stott (AJ)

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

William Esmieu (W)

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

Huw D Vater (HD)

Discovery from Charles River, Charles River, Chesterford Research Park, Saffron Walden CB10 1XL, U.K.
ORCID: 0000-0002-2904-1848

Stephen J Webster (SJ)

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

Amanda J Van de Poël (AJ)

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

Cole Clissold (C)

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

Brett Cosgrove (B)

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

Benjamin Sutton (B)

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

Jonathan A Spencer (JA)

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

Perla Breccia (P)

Discovery from Charles River, Charles River, Chesterford Research Park, Saffron Walden CB10 1XL, U.K.
ORCID: 0000-0002-8552-9012

Emanuela Gancia (E)

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

Silvia Bonomo (S)

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

Tammy Ladduwahetty (T)

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

Ovadia Lazari (O)

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

Hiral Patel (H)

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

Helen C Atton (HC)

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

Steve Clifton (S)

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

Daniel M Mota (DM)

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

Dario Magnani (D)

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

Amy O'Neill (A)

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

Marta Stebbeds (M)

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

Natsuko Macabuag (N)

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

Daniel Todd (D)

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

Maria E Herva (ME)

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

Philip Mitchell (P)

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

Mijke Visser (M)

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

Sara Compte Sancerni (S)

Charles River, Darwinweg 24, 2333 CR Leiden, The Netherlands.
ORCID: 0000-0003-2297-7368

Laure Grand Moursel (L)

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

Marta da Silva (M)

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

Eva Kritikou (E)

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

Taneli T Heikkinen (TT)

Charles River, Microkatu 1, Kuopio 70210 Finland.

Tamuna Bolkvadze (T)

Charles River, Microkatu 1, Kuopio 70210 Finland.

Valentina Fodale (V)

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

Debora Spadafora (D)

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

Manuel Daldin (M)

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

Alberto Bresciani (A)

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

John E Mangette (JE)

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

Elizabeth M Doherty (EM)

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

Matthew R Lee (MR)

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

Todd Herbst (T)

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

Edith Monteagudo (E)

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

Douglas Macdonald (D)

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

Nikolay V Plotnikov (NV)

CHDI Management/CHDI Foundation, 6080 Center Drive, Los Angeles, California 90045, United States.
ORCID: 0000-0003-2341-4663

Mark Chambers (M)

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

George McAllister (G)

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

Ignacio Muňoz-Sanjuan (I)

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

Celia Dominguez (C)

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

Classifications MeSH