TY - JOUR

T1 - AAV-based vectors for human diseases modeling in laboratory animals

AU - Aliev, Timur I.

AU - Yudkin, Dmitry V.

N1 - The author(s) declare that financial support was received for the research, authorship, and/or publication of this article. This research work was supported by the Academic leadership program Priority 2030 proposed by Federal 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).

PY - 2025/2/12

Y1 - 2025/2/12

N2 - 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.

AB - 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.

KW - adeno-associated virus

KW - animal models

KW - gene therapy

KW - human diseases

KW - transgenesis

UR - https://www.mendeley.com/catalogue/74103905-4f77-3e26-a2eb-73b3b1a5e274/

UR - https://www.scopus.com/record/display.uri?eid=2-s2.0-85218634353&origin=inward&txGid=b11d7678402510cf6e039e27f5335cbd

U2 - 10.3389/fmed.2024.1499605

DO - 10.3389/fmed.2024.1499605

M3 - Article

C2 - 40007819

VL - 11

JO - Frontiers in Medicine

JF - Frontiers in Medicine

SN - 2296-858X

M1 - 1499605

ER -