| Title: |
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| Authors: |
Giulia Massaro, Amy F. Geard, Wenfei Liu, Oliver Coombe-Tennant, Simon N. Waddington, Julien Baruteau, Paul Gissen, Ahad A. Rahim |
| Source: |
Biomolecules, Vol 11, Iss 4, p 611 (2021) |
| Publisher Information: |
MDPI AG, 2021. |
| Publication Year: |
2021 |
| Collection: |
LCC:Microbiology |
| Subject Terms: |
lysosomal diseases, gene therapy, viral vectors, Microbiology, QR1-502 |
| More Details: |
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. |
| Document Type: |
article |
| File Description: |
electronic resource |
| Language: |
English |
| ISSN: |
2218-273X |
| Relation: |
https://www.mdpi.com/2218-273X/11/4/611; https://doaj.org/toc/2218-273X |
| DOI: |
10.3390/biom11040611 |
| Access URL: |
https://doaj.org/article/0d64169604c944538a096cbb0b3f671b |
| Accession Number: |
edsdoj.0d64169604c944538a096cbb0b3f671b |
| Database: |
Directory of Open Access Journals |
