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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development. (2021)

First Author: Massaro G

Attributed to: Phase I/II clinical trial evaluating AAV-mediated gene therapy for a severe paediatric metabolic liver disease: Ornithine Transcarbamylase deficiency funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.3390/biom11040611

PubMed Identifier: 33924076

Publication URI: http://europepmc.org/abstract/MED/33924076

Type: Journal Article/Review

Volume: 11

Parent Publication: Biomolecules

Issue: 4

ISSN: 2218-273X