ELX-02 Generates Protein via Premature Stop Codon Read-Through without Inducing Native Stop Codon Read-Through Proteins.


Journal

The Journal of pharmacology and experimental therapeutics
ISSN: 1521-0103
Titre abrégé: J Pharmacol Exp Ther
Pays: United States
ID NLM: 0376362

Informations de publication

Date de publication:
08 2020
Historique:
received: 07 02 2020
accepted: 30 04 2020
pubmed: 8 5 2020
medline: 13 11 2020
entrez: 8 5 2020
Statut: ppublish

Résumé

ELX-02 is a clinical stage, small-molecule eukaryotic ribosomal selective glycoside acting to induce read-through of premature stop codons (PSCs) that results in translation of full-length protein. However, improved read-through at PSCs has raised the question of whether native stop codon (NSC) fidelity would be impacted. Here, we compare read-through by ELX-02 in PSC and NSC contexts. DMS-114 cells containing a PSC in the

Identifiants

pubmed: 32376628
pii: jpet.120.265595
doi: 10.1124/jpet.120.265595
doi:

Substances chimiques

Codon, Terminator 0
ELX-02 0
Furans 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

264-272

Informations de copyright

Copyright © 2020 The Author(s).

Auteurs

Daniel K Crawford (DK)

Eloxx Pharmaceuticals, Inc., Waltham, Massachusetts.

Iris Alroy (I)

Eloxx Pharmaceuticals, Inc., Waltham, Massachusetts.

Neal Sharpe (N)

Eloxx Pharmaceuticals, Inc., Waltham, Massachusetts.

Matthew M Goddeeris (MM)

Eloxx Pharmaceuticals, Inc., Waltham, Massachusetts matthew.goddeeris@eloxxpharma.com.

Greg Williams (G)

Eloxx Pharmaceuticals, Inc., Waltham, Massachusetts.

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Classifications MeSH