Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.

adeno-associated viral vectors breath test classic galactosemia galactose oxidation gene therapy lipid nanoparticles mRNA-based therapy

Journal

Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857

Informations de publication

Date de publication:
14 Mar 2024
Historique:
received: 13 09 2023
accepted: 12 01 2024
medline: 14 2 2024
pubmed: 14 2 2024
entrez: 14 2 2024
Statut: epublish

Résumé

Despite the implementation of lifesaving newborn screening programs and a galactose-restricted diet, many patients with classic galactosemia develop long-term debilitating neurological deficits and primary ovarian insufficiency. Previously, we showed that the administration of human

Identifiants

DOI: 10.1016/j.omtm.2024.101191 PMID: 38352271 PMC: PMC10863324
pubmed: 38352271
doi: 10.1016/j.omtm.2024.101191
pii: S2329-0501(24)00007-X
pmc: PMC10863324
doi:

Types de publication

Journal Article

Langues

eng

Pagination

101191

Informations de copyright

© 2024 The Author(s).

Déclaration de conflit d'intérêts

The authors declare the following competing interests: X.Y., F.W., J.T., L.C., R.D., S.C., S.M., Y.W., M.Z., J.S., P.G.V.M., and P.F.F. are employees of Moderna and hold equities from the company.

Auteurs

Bijina Balakrishnan (B)

Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA.

Xinhua Yan (X)

Moderna, Cambridge, MA 02139, USA.

Marshall D McCue (MD)

Sable Systems International, North Las Vegas, NV 89032, USA.

Olivia Bellagamba (O)

Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA.

Aaron Guo (A)

Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA.

Felicity Winkler (F)

Moderna, Cambridge, MA 02139, USA.

Jason Thall (J)

Moderna, Cambridge, MA 02139, USA.

Lisa Crawford (L)

Moderna, Cambridge, MA 02139, USA.

Rain Dimen (R)

Moderna, Cambridge, MA 02139, USA.

Sara Chen (S)

Moderna, Cambridge, MA 02139, USA.

Sean McEnaney (S)

Moderna, Cambridge, MA 02139, USA.

Yiman Wu (Y)

Moderna, Cambridge, MA 02139, USA.

Mike Zimmer (M)

Moderna, Cambridge, MA 02139, USA.

Joe Sarkis (J)

Moderna, Cambridge, MA 02139, USA.

Paolo G V Martini (PGV)

Moderna, Cambridge, MA 02139, USA.

Patrick F Finn (PF)

Moderna, Cambridge, MA 02139, USA.

Kent Lai (K)

Division of Medical Genetics, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT 84108, USA.

Classifications MeSH