Advances in HIV Gene Therapy
Early gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, leading to the appr...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2024-02-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/25/5/2771 |
| _version_ | 1797264413462364160 |
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| author | Rose Kitawi Scott Ledger Anthony D. Kelleher Chantelle L. Ahlenstiel |
| author_facet | Rose Kitawi Scott Ledger Anthony D. Kelleher Chantelle L. Ahlenstiel |
| author_sort | Rose Kitawi |
| collection | DOAJ |
| description | Early gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy product targeting genetic mutations in 2017. Gene therapy (GT) can be delivered either in vivo or ex vivo. An ex vivo approach to gene therapy is advantageous, as it allows for the characterization of the gene-modified cells and the selection of desired properties before patient administration. Autologous cells can also be used during this process which eliminates the possibility of immune rejection. This review highlights the various stages of ex vivo gene therapy, current research developments that have increased the efficiency and safety of this process, and a comprehensive summary of Human Immunodeficiency Virus (HIV) gene therapy studies, the majority of which have employed the ex vivo approach. |
| first_indexed | 2024-04-25T00:28:30Z |
| format | Article |
| id | doaj.art-0f224dc24c0d4e8284c9133e28bf9643 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-04-25T00:28:30Z |
| publishDate | 2024-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-0f224dc24c0d4e8284c9133e28bf96432024-03-12T16:46:15ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672024-02-01255277110.3390/ijms25052771Advances in HIV Gene TherapyRose Kitawi0Scott Ledger1Anthony D. Kelleher2Chantelle L. Ahlenstiel3Kirby Institute, University of New South Wales, Kensington, NSW 2052, AustraliaKirby Institute, University of New South Wales, Kensington, NSW 2052, AustraliaKirby Institute, University of New South Wales, Kensington, NSW 2052, AustraliaKirby Institute, University of New South Wales, Kensington, NSW 2052, AustraliaEarly gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy product targeting genetic mutations in 2017. Gene therapy (GT) can be delivered either in vivo or ex vivo. An ex vivo approach to gene therapy is advantageous, as it allows for the characterization of the gene-modified cells and the selection of desired properties before patient administration. Autologous cells can also be used during this process which eliminates the possibility of immune rejection. This review highlights the various stages of ex vivo gene therapy, current research developments that have increased the efficiency and safety of this process, and a comprehensive summary of Human Immunodeficiency Virus (HIV) gene therapy studies, the majority of which have employed the ex vivo approach.https://www.mdpi.com/1422-0067/25/5/2771ex vivogene therapystem cellsvectorHIV |
| spellingShingle | Rose Kitawi Scott Ledger Anthony D. Kelleher Chantelle L. Ahlenstiel Advances in HIV Gene Therapy International Journal of Molecular Sciences ex vivo gene therapy stem cells vector HIV |
| title | Advances in HIV Gene Therapy |
| title_full | Advances in HIV Gene Therapy |
| title_fullStr | Advances in HIV Gene Therapy |
| title_full_unstemmed | Advances in HIV Gene Therapy |
| title_short | Advances in HIV Gene Therapy |
| title_sort | advances in hiv gene therapy |
| topic | ex vivo gene therapy stem cells vector HIV |
| url | https://www.mdpi.com/1422-0067/25/5/2771 |
| work_keys_str_mv | AT rosekitawi advancesinhivgenetherapy AT scottledger advancesinhivgenetherapy AT anthonydkelleher advancesinhivgenetherapy AT chantellelahlenstiel advancesinhivgenetherapy |