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adeno-associated virus serotype 4
- Development stage
- Preclinical
- Lead developer
- Pluriomics
- Modality
- Gene Therapies
- Administration
- Intravenous, Intracerebroventricular, Intravitreal, Intramuscular
01
Overview
Adeno-associated virus serotype 4 (AAV4) is a viral vector platform utilized in gene therapy to deliver therapeutic genetic material to specific tissues. Derived from the Parvoviridae family, AAV4 is characterized by its unique cellular tropism, which is mediated by binding to O-linked sialic acid on the surface of target cells. It has shown particular efficiency in transducing the ependymal lining of the ventricles in the central nervous system and the retinal pigmented epithelium in the eye. In comparative studies for cardiac gene delivery, AAV4 has demonstrated lower transduction efficiency in cardiomyocytes compared to other serotypes like AAV1, AAV6, or AAV9. As a replication-deficient vector, it is engineered to provide long-term transgene expression with a low immunogenic profile.
- Other names
- adeno-associated virus 4
02
Targets
SA (Sialic acid)
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