AAV genome modification for efficient AAV production

Walaa Asaad; Polina Volos; Denis Maksimov; Elena Khavina; Andrei Deviatkin; Olga Mityaeva; Pavel Volchkov

doi:10.1016/j.heliyon.2023.e15071

AAV genome modification for efficient AAV production

Heliyon. 2023 Apr 1;9(4):e15071. doi: 10.1016/j.heliyon.2023.e15071. eCollection 2023 Apr.

Authors

Walaa Asaad¹, Polina Volos¹, Denis Maksimov¹, Elena Khavina¹, Andrei Deviatkin¹, Olga Mityaeva¹, Pavel Volchkov¹

Affiliation

¹ Genome Engineering Laboratory, Moscow Institute of Physics and Technology, Dolgoprudniy, Russian Federation.

Abstract

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.

Keywords: AAP; AAV; AAV production; ITR; ITRs; MAAP.

Publication types

Review