In vivo applications and toxicities of AAV-based gene therapies in rare diseases
Abstract Adeno-associated virus (AAV), renowned for its exceptionally low pathogenicity and significant efficacy in clinical gene therapy, has emerged as a leading delivery vector in the field of gene therapy. AAV can achieve stable gene expression in various tissues, which has made it a promising t...
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| Format: | Article |
| Language: | English |
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BMC
2025-07-01
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| Series: | Orphanet Journal of Rare Diseases |
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| Online Access: | https://doi.org/10.1186/s13023-025-03893-z |
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| _version_ | 1849388914999033856 |
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| author | Qian Zhao Huifang Peng Yujin Ma Huijun Yuan Hongwei Jiang |
| author_facet | Qian Zhao Huifang Peng Yujin Ma Huijun Yuan Hongwei Jiang |
| author_sort | Qian Zhao |
| collection | DOAJ |
| description | Abstract Adeno-associated virus (AAV), renowned for its exceptionally low pathogenicity and significant efficacy in clinical gene therapy, has emerged as a leading delivery vector in the field of gene therapy. AAV can achieve stable gene expression in various tissues, which has made it a promising treatment for genetic disorders. To date, eight AAV-based gene therapies have been approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). This review summarizes clinical trials of AAV gene therapies for rare diseases, including ophthalmic diseases, nervous system disorders, hematological diseases, neuromuscular diseases, lysosomal storage diseases. We also explore potential side effects and toxicities associated with AAV therapies. Our objective is to provide valuable insights for researchers and clinicians working on AAV-based therapies, helping improve the safety and effectiveness of these treatments. |
| format | Article |
| id | doaj-art-1dfd4295da05466ebe4b6e8b4d0f3efb |
| institution | Kabale University |
| issn | 1750-1172 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj-art-1dfd4295da05466ebe4b6e8b4d0f3efb2025-08-20T03:42:07ZengBMCOrphanet Journal of Rare Diseases1750-11722025-07-0120111410.1186/s13023-025-03893-zIn vivo applications and toxicities of AAV-based gene therapies in rare diseasesQian Zhao0Huifang Peng1Yujin Ma2Huijun Yuan3Hongwei Jiang4Luoyang Key Laboratory of Clinical Multiomics and Translational Medicine, Henan Key Laboratory of Rare Diseases, Endocrinology and Metabolism Center, The First Affiliated Hospital, College of Clinical Medicine, Henan University of Science and TechnologyLuoyang Key Laboratory of Clinical Multiomics and Translational Medicine, Henan Key Laboratory of Rare Diseases, Endocrinology and Metabolism Center, The First Affiliated Hospital, College of Clinical Medicine, Henan University of Science and TechnologyLuoyang Key Laboratory of Clinical Multiomics and Translational Medicine, Henan Key Laboratory of Rare Diseases, Endocrinology and Metabolism Center, The First Affiliated Hospital, College of Clinical Medicine, Henan University of Science and TechnologyInstitute of Rare Diseases, West China Hospital of Sichuan UniversityLuoyang Key Laboratory of Clinical Multiomics and Translational Medicine, Henan Key Laboratory of Rare Diseases, Endocrinology and Metabolism Center, The First Affiliated Hospital, College of Clinical Medicine, Henan University of Science and TechnologyAbstract Adeno-associated virus (AAV), renowned for its exceptionally low pathogenicity and significant efficacy in clinical gene therapy, has emerged as a leading delivery vector in the field of gene therapy. AAV can achieve stable gene expression in various tissues, which has made it a promising treatment for genetic disorders. To date, eight AAV-based gene therapies have been approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). This review summarizes clinical trials of AAV gene therapies for rare diseases, including ophthalmic diseases, nervous system disorders, hematological diseases, neuromuscular diseases, lysosomal storage diseases. We also explore potential side effects and toxicities associated with AAV therapies. Our objective is to provide valuable insights for researchers and clinicians working on AAV-based therapies, helping improve the safety and effectiveness of these treatments.https://doi.org/10.1186/s13023-025-03893-zAdeno-associated virusClinical trialsEfficacyAdverse effects/ToxicitiesGene therapies |
| spellingShingle | Qian Zhao Huifang Peng Yujin Ma Huijun Yuan Hongwei Jiang In vivo applications and toxicities of AAV-based gene therapies in rare diseases Orphanet Journal of Rare Diseases Adeno-associated virus Clinical trials Efficacy Adverse effects/Toxicities Gene therapies |
| title | In vivo applications and toxicities of AAV-based gene therapies in rare diseases |
| title_full | In vivo applications and toxicities of AAV-based gene therapies in rare diseases |
| title_fullStr | In vivo applications and toxicities of AAV-based gene therapies in rare diseases |
| title_full_unstemmed | In vivo applications and toxicities of AAV-based gene therapies in rare diseases |
| title_short | In vivo applications and toxicities of AAV-based gene therapies in rare diseases |
| title_sort | in vivo applications and toxicities of aav based gene therapies in rare diseases |
| topic | Adeno-associated virus Clinical trials Efficacy Adverse effects/Toxicities Gene therapies |
| url | https://doi.org/10.1186/s13023-025-03893-z |
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