Adenoassoziierte

Adenoassoziierte is an adjective used in molecular biology and virology to denote a relationship with adenoviruses or, more commonly, with adeno-associated viruses. In German literature it is often encountered in connection with the term adenoassoziiertes Virus (AAV), a small virus used as a vector in gene delivery and basic research.

Adenoassoziierte Viren belong to the Parvoviridae family, genus Dependoparvovirus. They are non-enveloped, with a single-stranded DNA genome of about 4.7 kilobases. In nature AAV replication depends on helper functions provided by a co-infecting adenovirus or herpesvirus; recombinant AAV vectors used in research and therapy are engineered to be replication-deficient and to carry a therapeutic gene, while helper functions are supplied in trans during production.

AAVs exhibit serotype diversity (for example AAV1–AAV9), which influences tissue tropism and transduction efficiency. The packaging capacity is limited, constraining the size of the therapeutic gene and regulatory elements. They typically persist as episomes in non-dividing cells, enabling long-term expression in some tissues with a relatively favorable safety profile. However, pre-existing immunity to common serotypes and potential immunogenicity of the capsid can affect effectiveness.

Production and application: AAV vectors are produced in helper-free systems using plasmids that provide essential functions in trans. In research and clinical contexts they are used for gene delivery to restore or modify gene expression. Several therapies approved or in development employ AAV vectors, including treatments for retinal dystrophies and spinal muscular atrophy, reflecting the role of adeno-associated vectors in modern gene therapy.