AAV-based vectors for human diseases modeling in laboratory animals

The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models. The advantage of transgenic animals lies in their ability to simulate specific pro...

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Main Authors: Timur I. Aliev, Dmitry V. Yudkin
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-02-01
Series:Frontiers in Medicine
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Online Access:https://www.frontiersin.org/articles/10.3389/fmed.2024.1499605/full
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author Timur I. Aliev
Dmitry V. Yudkin
author_facet Timur I. Aliev
Dmitry V. Yudkin
author_sort Timur I. Aliev
collection DOAJ
description The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models. The advantage of transgenic animals lies in their ability to simulate specific properties desired by researchers. However, there is often a need for the rapid production of transgenic animal models, especially in situations like a pandemic, as was evident during COVID-19. An important tool for transgenesis is the adeno-associated virus. The genome of adeno-associated virus serves as a convenient expression cassette for delivering various DNA constructs into cells, and this method has proven effective in practice. This review analyzes the features of the adeno-associated virus genome that make it an advantageous vector for transgenesis. Additionally, examples of utilizing adeno-associated viral vectors to create animal models for hereditary, oncological, and viral human diseases are provided.
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publisher Frontiers Media S.A.
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spelling doaj-art-f19d384675cd4e7882925733ca19f2612025-02-12T04:10:58ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2025-02-011110.3389/fmed.2024.14996051499605AAV-based vectors for human diseases modeling in laboratory animalsTimur I. Aliev0Dmitry V. Yudkin1Department of Natural Sciences, Novosibirsk State University, Novosibirsk, RussiaFederal State Autonomous Educational Institution of Higher Education I.M. Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation (Sechenov University), Moscow, RussiaThe development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models. The advantage of transgenic animals lies in their ability to simulate specific properties desired by researchers. However, there is often a need for the rapid production of transgenic animal models, especially in situations like a pandemic, as was evident during COVID-19. An important tool for transgenesis is the adeno-associated virus. The genome of adeno-associated virus serves as a convenient expression cassette for delivering various DNA constructs into cells, and this method has proven effective in practice. This review analyzes the features of the adeno-associated virus genome that make it an advantageous vector for transgenesis. Additionally, examples of utilizing adeno-associated viral vectors to create animal models for hereditary, oncological, and viral human diseases are provided.https://www.frontiersin.org/articles/10.3389/fmed.2024.1499605/fulladeno-associated virustransgenesisanimal modelshuman diseasesgene therapy
spellingShingle Timur I. Aliev
Dmitry V. Yudkin
AAV-based vectors for human diseases modeling in laboratory animals
Frontiers in Medicine
adeno-associated virus
transgenesis
animal models
human diseases
gene therapy
title AAV-based vectors for human diseases modeling in laboratory animals
title_full AAV-based vectors for human diseases modeling in laboratory animals
title_fullStr AAV-based vectors for human diseases modeling in laboratory animals
title_full_unstemmed AAV-based vectors for human diseases modeling in laboratory animals
title_short AAV-based vectors for human diseases modeling in laboratory animals
title_sort aav based vectors for human diseases modeling in laboratory animals
topic adeno-associated virus
transgenesis
animal models
human diseases
gene therapy
url https://www.frontiersin.org/articles/10.3389/fmed.2024.1499605/full
work_keys_str_mv AT timurialiev aavbasedvectorsforhumandiseasesmodelinginlaboratoryanimals
AT dmitryvyudkin aavbasedvectorsforhumandiseasesmodelinginlaboratoryanimals