Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
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 r...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2021-04-01
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| Series: | Biomolecules |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2218-273X/11/4/611 |
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| author | Giulia Massaro Amy F. Geard Wenfei Liu Oliver Coombe-Tennant Simon N. Waddington Julien Baruteau Paul Gissen Ahad A. Rahim |
| author_facet | Giulia Massaro Amy F. Geard Wenfei Liu Oliver Coombe-Tennant Simon N. Waddington Julien Baruteau Paul Gissen Ahad A. Rahim |
| author_sort | Giulia Massaro |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T12:09:17Z |
| format | Article |
| id | doaj.art-0d64169604c944538a096cbb0b3f671b |
| institution | Directory Open Access Journal |
| issn | 2218-273X |
| language | English |
| last_indexed | 2024-03-10T12:09:17Z |
| publishDate | 2021-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomolecules |
| spelling | doaj.art-0d64169604c944538a096cbb0b3f671b2023-11-21T16:19:43ZengMDPI AGBiomolecules2218-273X2021-04-0111461110.3390/biom11040611Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical DevelopmentGiulia Massaro0Amy F. Geard1Wenfei Liu2Oliver Coombe-Tennant3Simon N. Waddington4Julien Baruteau5Paul Gissen6Ahad A. Rahim7UCL School of Pharmacy, University College London, London WC1N 1AX, UKUCL School of Pharmacy, University College London, London WC1N 1AX, UKUCL School of Pharmacy, University College London, London WC1N 1AX, UKUCL School of Pharmacy, University College London, London WC1N 1AX, UKWits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2193, South AfricaMetabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 1EH, UKGreat Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, National Institute of Health Research, University College London, London WC1N 1EH, UKUCL School of Pharmacy, University College London, London WC1N 1AX, UKRare 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.https://www.mdpi.com/2218-273X/11/4/611lysosomal diseasesgene therapyviral vectors |
| spellingShingle | Giulia Massaro Amy F. Geard Wenfei Liu Oliver Coombe-Tennant Simon N. Waddington Julien Baruteau Paul Gissen Ahad A. Rahim Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development Biomolecules lysosomal diseases gene therapy viral vectors |
| title | Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| title_full | Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| title_fullStr | Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| title_full_unstemmed | Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| title_short | Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development |
| title_sort | gene therapy for lysosomal storage disorders ongoing studies and clinical development |
| topic | lysosomal diseases gene therapy viral vectors |
| url | https://www.mdpi.com/2218-273X/11/4/611 |
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