Massaro, G.Geard, A.F.Liu, W.Coombe-Tennant, O.Waddington, S.N.Baruteau, J.Gissen, P.Rahim, A.A.2024-06-022024-06-022021-04-20Massaro G, Geard AF, Liu W, Coombe-Tennant O, Waddington SN, Baruteau J, Gissen P, Rahim AA. Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development. Biomolecules. 2021 Apr 20;11(4):611. doi: 10.3390/biom11040611.10.3390/biom1104061110.3390/biom11040611https://pubmed.ncbi.nlm.nih.gov/33924076/https://hdl.handle.net/11288/596209Rare monogenic disorders such as lysosomal diseases have been at the forefront in the development of novel treatments where therapeutic options are either limited or unavailable. The increasing number of successful pre-clinical and clinical studies in the last decade demonstrates that gene therapy represents a feasible option to address the unmet medical need of these patients. This article provides a comprehensive overview of the current state of the field, reviewing the most used viral gene delivery vectors in the context of lysosomal storage disorders, a selection of relevant pre-clinical studies and ongoing clinical trials within recent years.enAttribution 3.0 United Stateshttp://creativecommons.org/licenses/by/3.0/us/Lysosomal diseasesGene therapyViral vectorsGene EditingSDG-03 Good health and well-beingArticleBiomolecules