Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".
Deadline for manuscript submissions: 30 June 2026
Special Issue Editors
Interests: nanofabrication approaches for oral drug delivery; electrospinning; solid dispersions; physical characterization techniques; microfluidics; nanogels for gene delivery; taste masked dosage forms
Special Issues, Collections and Topics in MDPI journals
Interests: pharmacology and pharmaceutical sciences; biochemistry and cell biology; gene and molecular therapy; neurosciences; microfluidics and nanofluidics
Special Issue Information
Dear Colleagues,
Adeno-associated virus (AAV) has emerged as one of the most important vectors in modern gene therapy, enabling long-lasting, tissue-targeted delivery with a strong safety profile. AAV gene therapy has revolutionised treatment for some diseases, but remains limited by its high cost, variable efficacy, and translational uncertainty. As clinical demand for AAV grows, advancing its bioprocessing and engineering is essential to overcome its current limitations in terms of potency, specificity, manufacturability, and scalability. This Special Issue of Pharmaceutics highlights innovations in AAV engineering and manufacturing, from designing next-generation capsids with enhanced tropism and transduction efficiency and reduced immunogenicity, to optimising upstream production systems and downstream purification workflows that maximise yield and product quality. We welcome studies that apply synthetic biology, high-throughput screening, and computational design to engineer improved vectors, as well as work that develops robust analytical tools, stable formulations, and regulatory-ready CMC strategies. By integrating biological insight with bioengineering advances, this Special Issue aims to accelerate the development of more effective, scalable, and accessible AAV-based therapeutics.
Prof. Dr. Duncan Craig
Dr. Giulia Massaro
Guest Editors
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Keywords
- AAV
- gene therapy
- viral vector
- capsid engineering
- bioprocessing
- synthetic biology
- computational design
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