AAV genome modification for efficient AAV production

The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require usi...

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Main Authors: Walaa Asaad, Polina Volos, Denis Maksimov, Elena Khavina, Andrei Deviatkin, Olga Mityaeva, Pavel Volchkov
Format: Article
Language:English
Published: Elsevier 2023-04-01
Series:Heliyon
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2405844023022788
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author Walaa Asaad
Polina Volos
Denis Maksimov
Elena Khavina
Andrei Deviatkin
Olga Mityaeva
Pavel Volchkov
author_facet Walaa Asaad
Polina Volos
Denis Maksimov
Elena Khavina
Andrei Deviatkin
Olga Mityaeva
Pavel Volchkov
author_sort Walaa Asaad
collection DOAJ
description The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require using functional, pure, and high titers of vector particles. In fact, the current knowledge of AAV structure and genome helps improve the scalable production of AAV vectors. In this review, we summarize the latest studies on the optimization of scalable AAV production through modifying the AAV genome or biological processes inside the cell.
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spelling doaj.art-d3c5cf6e88474a2886297eee5efe51862023-04-29T14:55:10ZengElsevierHeliyon2405-84402023-04-0194e15071AAV genome modification for efficient AAV productionWalaa Asaad0Polina Volos1Denis Maksimov2Elena Khavina3Andrei Deviatkin4Olga Mityaeva5Pavel Volchkov6Corresponding author.; Genome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationGenome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian FederationThe adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require using functional, pure, and high titers of vector particles. In fact, the current knowledge of AAV structure and genome helps improve the scalable production of AAV vectors. In this review, we summarize the latest studies on the optimization of scalable AAV production through modifying the AAV genome or biological processes inside the cell.http://www.sciencedirect.com/science/article/pii/S2405844023022788AAVAAV productionITRITRsAAPMAAP
spellingShingle Walaa Asaad
Polina Volos
Denis Maksimov
Elena Khavina
Andrei Deviatkin
Olga Mityaeva
Pavel Volchkov
AAV genome modification for efficient AAV production
Heliyon
AAV
AAV production
ITR
ITRs
AAP
MAAP
title AAV genome modification for efficient AAV production
title_full AAV genome modification for efficient AAV production
title_fullStr AAV genome modification for efficient AAV production
title_full_unstemmed AAV genome modification for efficient AAV production
title_short AAV genome modification for efficient AAV production
title_sort aav genome modification for efficient aav production
topic AAV
AAV production
ITR
ITRs
AAP
MAAP
url http://www.sciencedirect.com/science/article/pii/S2405844023022788
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