Targeting AGAT gene expression - a drug screening approach for the treatment of GAMT deficiency.

There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

Abstract

Targeting the enzyme L-Arginine:glycine amidinotransferase (AGAT) to reduce the formation of guanidinoacetate (GAA) in patients with guanidinoacetate methyltransferase (GAMT) deficiency, we attempted to identify drugs for repurposing that reduce the expression of AGAT via transcriptional inhibition.

Related collections

Author and article information

Journal

Journal ID (iso-abbrev): Expert Opin Drug Discov

Title: Expert opinion on drug discovery

Publisher: Informa UK Limited

ISSN (Electronic): 1746-045X

ISSN (Print): 1746-0441

Publication date (Electronic): Nov 2024

Volume: 19

Issue: 11

Affiliations

[1 ] Genetics and Genome Biology, The Hospital for Sick Children, Toronto, ON, Canada.

[2 ] Department of Biochemistry, University of Toronto, Toronto, ON, Canada.

[3 ] Samuel Lunenfeld Research Institute, Mount Sinai Hospital, Toronto, ON, Canada.

[4 ] Clinical Pharmacology and Toxicology, The Hospital for Sick Children, Toronto, ON, Canada.

[5 ] Department of Pediatrics, University of Toronto, Toronto, ON, Canada.

Article

DOI: 10.1080/17460441.2024.2412994

PubMed ID: 39402976

SO-VID: c8228e7e-cd1e-42f9-9e20-5d22f254cdab

History

Keywords: AGAT gene repression,GAMT deficiency,HAP1 AGAT gene NLuc reporter fusion,HeLa AGAT promoter FLuc reporter cells,drug screening

Data availability:

Keywords: AGAT gene repression, GAMT deficiency, HAP1 AGAT gene NLuc reporter fusion, HeLa AGAT promoter FLuc reporter cells, drug screening

Comments

Comment on this article

scite_