Work

In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model

Year: 2025

Type: article

Abstract: direct LV gene therapy as a promising approach for treating MPS IVA and similar skeletal disorders.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2025.101514

Authors: Betul Celik, Estera Rintz, Shaukat Khan, Andrés Felipe Leal, Fnu Nidhi, Shunji Tomatsu

Institutions: Nemours Children's Health System, University of Delaware, University of Gdańsk, Thomas Jefferson University, Pontificia Universidad Javeriana

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900

Funders: National MPS Society, Bennett Foundation, National Institutes of Health, National Institute of Child Health and Human Development, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Carol Ann Foundation

Awards: G3350067605, G5843161533

OpenAlex ID: W4411334583

FWCI: 1.645

Cites: 85

Cited by: 3

Related to: 10