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Review
. 2021 Apr 20;11(4):611.
doi: 10.3390/biom11040611.

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Giulia Massaro  1 , Amy F Geard  1   2 , Wenfei Liu  1 , Oliver Coombe-Tennant  1 , Simon N Waddington  2   3 , Julien Baruteau  4   5 , Paul Gissen  5 , Ahad A Rahim  1
Affiliations
Review

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Giulia Massaro et al. Biomolecules. .

Abstract

Rare 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.

Keywords: gene therapy; lysosomal diseases; viral vectors.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Schematic representation of the vector genomes (LTR: Long Terminal Repeat; Ψ: packaging signal; GOI: Gene of Interest; cPPT: central Polypurine Tract; RRE: rev-binding element; WPRE: Woodchuck Post-transcriptional Regulatory Element; ITR: Inverted Terminal Repeat; L: Late adenoviral genes; E: Early adenoviral genes).
Figure 2
Figure 2
Main routes of administration for in vivo gene delivery to LSDs patients.
See this image and copyright information in PMC

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