Challenges in Adeno-Associated Virus Gene Therapy

Recombinant adeno-associated virus (AAV) is widely recognized as the most promising viral vector for clinicalgene therapy. Despite fatal adverse events with FDA and EMA-approved AAV gene therapies, more AAV genetherapies are expected to be approved. A critical challenge in the field remains the production of sufficientvectors with optimal tropism to target specific tissues, compounded by the lack of clear prospects forstandardized quality assurance. AAV tropism is highly species-dependent, which limits the relevance ofpreclinical studies in small animals to human applications. Recent advancements in Artificial Intelligence (AI)and machine learning have begun to transform problem-solving approaches in AAV research. AI-drivenapplications, particularly in capsid design, offer promising solutions to these challenges. This emergingtechnology is poised to revolutionize not only vector development and production but also the treatmentsavailable to patients receiving AAV-based therapies.Acta Biochimica Polonica is planning a Special Issue devoted to AI-assisted vector improvements,manufacturing, and quality assurance to facilitate this field moving forward. We are soliciting original papers,review articles, and minireviews addressing AAV manufacturing. Preferred manuscripts include, but are notlimited to:•Either comprehensive or brief, focused review articles on AAV’s species barriers, manufacturing, orquality assurance• Studies of the species barriers of AAV tropism• Innovative methods to improve vector production and quality assurance• Applications of AI technology, including but not limited to capsid designFor authors, please review the journal's information regarding Article Types, Author Guidelines, and Publishing Fees, or direct any questions to the Editorial Office: abp@frontierspartnerships.org.Even though abstract submission is not mandatory, we encourage all interested researchers to submit a manuscript summary before submitting their manuscript. Manuscript summaries do not have to coincide with the final abstract of the manuscripts. Topic editors: Kazuhiro Oka, Estera Rintz Source: https://www.frontierspartnerships.org/research-topics/161/challenges-in-adeno-associated-virus-gene-therapy