The PROCEED study: A phase I/II dose-escalation investigation of systemic AAV9-based gene therapy for peripheral manifestations of Gaucher disease

Molecular Genetics and Metabolism 2026

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Authors: Mia A. Weaver, Özlem Göker-Alpan, Pilar Giraldo, Jennifer L. Cohen, Eugen Mengel, Jeff Booth, Sebastian Boland, Rachel Manthe-Gross, Daniel Hatch, Sarah Neuhaus
DOI: 10.1016/j.ymgme.2025.109664
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Glycogen Storage Diseases and Myoclonus

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109664–109664
Publication date
1 February 2026
Language
EN