Adeno-Associated Virus (AAV) for Gene Therapy
A special issue of Viruses (ISSN 1999-4915). This special issue belongs to the section "Human Virology and Viral Diseases".
Deadline for manuscript submissions: 30 September 2024 | Viewed by 2576
Special Issue Editor
Special Issue Information
Dear Colleagues,
Adeno-associated virus (AAV) based vector have arguably emerged as the leading gene therapy modality. But, despite the fact that currently five AAV gene therapy treatments are approved by the FDA and/or the EMA and a plethora of early and late-stage clinical trials are underway, formidable hurdles to the broadest application to treat inherited and acquired diseases with AAV gene therapy remain.
Among those hurdles are pre-existing, innate and acquired immunity. Innate and acquired immunity are strongly correlated with the vector dose administered. Unfortunately, since a significant number of cell types are difficult to transduce with currently available AAV serotypes/variants, high vector amounts need often to be administered. This is a particularly acute limitation when the vector must be administered systemically. The production of the large amounts of virus required is often challenging and inevitably expensive. Below is a (non-inclusive) list of areas that are of special interest for the current special guest issue of Viruses.
- Approaches to reduce the vector dose without negatively influencing transduction efficiencies (e.g., the development of viruses with a more favorable transduction profile, more efficient routes of administration, understanding the basic biology of AAVs – particular in vivo models etc.);
- Developing methods to determine the human tropism of AAV serotypes/variants;
- Identifying the best model(s) to study AAV tropism, based on knowledge gained by the ability to determine the human tropism;
- Reduction of an innate immune response upon vector administration;
- Reduction of an adaptive immune response due to treatment with AAV vectors;
- Approaches to mitigate the deleterious effect of pre-existing immunity against the AAV serotype/variant used;
- The development of methods that allow vector re-administration;
- AAV vector manufacturing platforms;
- AAV vector quality control.
Dr. Thomas Weber
Guest Editor
Manuscript Submission Information
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Keywords
- AAV gene therapy
- immune responses triggered by AAV vector administration
- AAV-Biology
- novel AAV variants
- AAV vector manufacturing and quality control
