Adenoviral Vectors for Gene Therapy of Hereditary Diseases
Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found thei...
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MDPI AG
2024-12-01
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| Series: | Biology |
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| Online Access: | https://www.mdpi.com/2079-7737/13/12/1052 |
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| author | Anna Muravyeva Svetlana Smirnikhina |
| author_facet | Anna Muravyeva Svetlana Smirnikhina |
| author_sort | Anna Muravyeva |
| collection | DOAJ |
| description | Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found their place in gene therapy for hereditary diseases. This review provides an overview of AdVs, their features, and clinical trials using them for gene replacement therapy in monogenic diseases and analyzes the reasons for the failures of these studies. Additionally, current research on the modification of AdVs to reduce immune responses and target delivery is discussed. |
| format | Article |
| id | doaj-art-97a992d98d234746bd23be8cc6e5e651 |
| institution | OA Journals |
| issn | 2079-7737 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biology |
| spelling | doaj-art-97a992d98d234746bd23be8cc6e5e6512025-08-20T02:00:55ZengMDPI AGBiology2079-77372024-12-011312105210.3390/biology13121052Adenoviral Vectors for Gene Therapy of Hereditary DiseasesAnna Muravyeva0Svetlana Smirnikhina1Laboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye, 1, 115522 Moscow, RussiaLaboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye, 1, 115522 Moscow, RussiaAdenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found their place in gene therapy for hereditary diseases. This review provides an overview of AdVs, their features, and clinical trials using them for gene replacement therapy in monogenic diseases and analyzes the reasons for the failures of these studies. Additionally, current research on the modification of AdVs to reduce immune responses and target delivery is discussed.https://www.mdpi.com/2079-7737/13/12/1052adenoviral vectorgene therapyhereditary diseasescystic fibrosisornithine transcarbamylase deficiency |
| spellingShingle | Anna Muravyeva Svetlana Smirnikhina Adenoviral Vectors for Gene Therapy of Hereditary Diseases Biology adenoviral vector gene therapy hereditary diseases cystic fibrosis ornithine transcarbamylase deficiency |
| title | Adenoviral Vectors for Gene Therapy of Hereditary Diseases |
| title_full | Adenoviral Vectors for Gene Therapy of Hereditary Diseases |
| title_fullStr | Adenoviral Vectors for Gene Therapy of Hereditary Diseases |
| title_full_unstemmed | Adenoviral Vectors for Gene Therapy of Hereditary Diseases |
| title_short | Adenoviral Vectors for Gene Therapy of Hereditary Diseases |
| title_sort | adenoviral vectors for gene therapy of hereditary diseases |
| topic | adenoviral vector gene therapy hereditary diseases cystic fibrosis ornithine transcarbamylase deficiency |
| url | https://www.mdpi.com/2079-7737/13/12/1052 |
| work_keys_str_mv | AT annamuravyeva adenoviralvectorsforgenetherapyofhereditarydiseases AT svetlanasmirnikhina adenoviralvectorsforgenetherapyofhereditarydiseases |