What type of study is this?

This is a Literature Review study.

August 26, 2025Open Access

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

Key Points

AAV-based gene therapy aims to treat various diseases, but faces significant challenges with immune responses, which could limit its effectiveness.
The production of AAV has been shown to struggle with low viral titers, impacting availability and efficacy for patients.
Observational analysis of current AAV approaches highlights the risk of insertional mutagenesis, which poses safety concerns in clinical applications.
Effective scale-up strategies are needed to address production limitations, ensuring adequate supply and safety in gene therapy applications.

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.

KI fragen

Bookmark

View Full Paper