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Volume 26
Issue 17
10.3390/ijms26178282
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This is an early access version, the complete PDF, HTML, and XML versions will be available soon.
Review

AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies

by
Daniil Moldavskii
1,
Zarema Gilazieva
1,
Alisa Fattakhova
1,
Valeriya Solovyeva
1,
Shaza Issa
2,
Albert Sufianov
3,4,
Galina Sufianova
5 and
Albert Rizvanov
1,6,*
1
Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan 420008, Russia
2
Department of Genetics and Biotechnology, St. Petersburg State University, St. Petersburg 199034, Russia
3
Department of Neurosurgery, Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation (Sechenov University), Moscow 119991, Russia
4
The Research and Educational Institute of Neurosurgery, Peoples’ Friendship University of Russia (RUDN), Moscow 117198, Russia
5
Department of Pharmacology, Tyumen State Medical University, Tyumen 625023, Russia
6
Division of Medical and Biological Sciences, Tatarstan Academy of Sciences, Kazan 420111, Russia
*
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2025, 26(17), 8282; https://doi.org/10.3390/ijms26178282
Submission received: 16 July 2025 / Revised: 18 August 2025 / Accepted: 22 August 2025 / Published: 26 August 2025
(This article belongs to the Special Issue Viruses as Therapeutic Tools: Medical and Biotechnological Applications)
Versions Notes

Abstract

Currently, the development of adeno-associated virus (AAV)-based gene therapy is a promising method for treating various diseases and is gaining increasing popularity. However, the use of AAV has certain drawbacks and faces limitations such as immune responses and an increased risk of insertional mutagenesis, which have not always been adequately considered in the context of AAV therapy. Moreover, a significant limitation for the application of AAV lies in the challenge of producing it in large quantities. This article discusses the use of AAV in treating various diseases, reviews AAV production approaches, highlights challenges with insufficient viral titers during production, and explores potential solutions at key stages of AAV drug production.
Keywords: gene therapy; AAV production; gene therapy vectors; high-titer AAV; AAV manufacturing gene therapy; AAV production; gene therapy vectors; high-titer AAV; AAV manufacturing

Share and Cite

MDPI and ACS Style

Moldavskii, D.; Gilazieva, Z.; Fattakhova, A.; Solovyeva, V.; Issa, S.; Sufianov, A.; Sufianova, G.; Rizvanov, A. AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies. Int. J. Mol. Sci. 2025, 26, 8282. https://doi.org/10.3390/ijms26178282

AMA Style

Moldavskii D, Gilazieva Z, Fattakhova A, Solovyeva V, Issa S, Sufianov A, Sufianova G, Rizvanov A. AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies. International Journal of Molecular Sciences. 2025; 26(17):8282. https://doi.org/10.3390/ijms26178282

Chicago/Turabian Style

Moldavskii, Daniil, Zarema Gilazieva, Alisa Fattakhova, Valeriya Solovyeva, Shaza Issa, Albert Sufianov, Galina Sufianova, and Albert Rizvanov. 2025. "AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies" International Journal of Molecular Sciences 26, no. 17: 8282. https://doi.org/10.3390/ijms26178282

APA Style

Moldavskii, D., Gilazieva, Z., Fattakhova, A., Solovyeva, V., Issa, S., Sufianov, A., Sufianova, G., & Rizvanov, A. (2025). AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies. International Journal of Molecular Sciences, 26(17), 8282. https://doi.org/10.3390/ijms26178282

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