Work

Phase 1/2 dose-finding study to evaluate systemic administration of an AAV9-based gene therapy for peripheral manifestation of Gaucher disease: The PROCEED study

Year: 2025

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2024.108873

Authors: Sarah Neuhaus, Mia Weaver, Ozlem Goker-Alpan, Jennifer L. Cohen, Pilar Giraldo, Jeff Booth, Lee Shaughnessy, Daniel Hatch, Olga Uspenskaya, Jeffrey Sevigny

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4406981879

FWCI: 0.548

Cites:

Cited by: 1

Related to: 10