Adeno-Associated Virus (AAV): Small Virus, Big Impact – Promising Gene Therapy Vectors for the 21st Century and Beyond
A special issue of Microorganisms (ISSN 2076-2607). This special issue belongs to the section "Virology".
Deadline for manuscript submissions: closed (30 November 2021) | Viewed by 9680
Special Issue Editor
Special Issue Information
Dear Colleagues,
Advances and improvements in adeno-associated virus (AAV) vector technologies have resulted in rapid expansion of their development for use in the clinic. Successes in clinical trials have demonstrated their effectiveness as a therapeutic gene delivery vehicle for a variety of monogenic disorders, many of which currently lack treatments or therapies. The human immune response presents a challenge in using these vectors for gene delivery and a better understanding of the interaction of the virus capsid with the components of the immune system guides the development of next-generation vectors. Studies focused on the role of the virus capsid and interactions with cellular components will also result in improvements in tissue targeting and transduction efficiency. More efficient tissue targeting and gene expression will result in lower required effective therapeutic doses and will provide safer treatments for patients. This Special Issue will highlight recent advances in AAV vectorology, novel AAV gene therapy vectors, and current progress in moving these vectors into the clinic for a variety of disorders.
This Special Issue of Microorganisms will be dedicated to the following themes: advances in AAV viral vectors for their development as therapeutic gene delivery vehicles for use in the clinic; modified AAV capsids for the purpose of specific tissue targeting, improved production and purification, or immune evasion; development of novel AAV vectors for clinical conditions; investigations of novel AAV serotypes or modified AAVs for use in the clinic; next-generation AAV vectors.
Dr. Kim Van Vliet
Guest Editor
Manuscript Submission Information
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Keywords
- AAV vectors
- adeno-associated virus (AAV)
- gene therapy
- AAV capsid structure
- parvovirus
- AAV serotype
- transduction