Durability of transgene expression after rAAV gene therapy
Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved..
Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the lives of patients with certain genetic disorders by increasing or restoring function to affected tissues. Following the initial establishment of transgene expression, it is unknown how long the therapeutic effect will last, although animal and emerging human data show that expression can be maintained for more than 10 years. The durability of therapeutic response is key to long-term treatment success, especially since immune responses to rAAV vectors may prevent re-dosing with the same therapy. This review explores the non-immunological and immunological processes that may limit or improve durability and the strategies that can be used to increase the duration of the therapeutic effect.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:30 |
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| Enthalten in: |
Molecular therapy : the journal of the American Society of Gene Therapy - 30(2022), 4 vom: 06. Apr., Seite 1364-1380 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Muhuri, Manish [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 11.04.2022 Date Revised 13.05.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.ymthe.2022.03.004 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the lives of patients with certain genetic disorders by increasing or restoring function to affected tissues. Following the initial establishment of transgene expression, it is unknown how long the therapeutic effect will last, although animal and emerging human data show that expression can be maintained for more than 10 years. The durability of therapeutic response is key to long-term treatment success, especially since immune responses to rAAV vectors may prevent re-dosing with the same therapy. This review explores the non-immunological and immunological processes that may limit or improve durability and the strategies that can be used to increase the duration of the therapeutic effect | ||
| 650 | 4 | |a Journal Article | |
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| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a animals | |
| 650 | 4 | |a gene expression | |
| 650 | 4 | |a gene expression regulation | |
| 650 | 4 | |a gene therapy | |
| 650 | 4 | |a gene transfer techniques | |
| 650 | 4 | |a genetic therapy/methods | |
| 650 | 4 | |a genetic vectors | |
| 650 | 4 | |a human genetics | |
| 650 | 4 | |a transgenes | |
| 700 | 1 | |a Levy, Daniel I |e verfasserin |4 aut | |
| 700 | 1 | |a Schulz, Martin |e verfasserin |4 aut | |
| 700 | 1 | |a McCarty, Douglas |e verfasserin |4 aut | |
| 700 | 1 | |a Gao, Guangping |e verfasserin |4 aut | |
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