Molecular Classification
Viral vector, Gene therapy vector
Other Names
AAV serotype 9, rAAV9
Disease Roles
Therapeutic agent for genetic diseasesTreatment of neurological disordersTreatment of cardiac disorders

Adeno-Associated Virus Serotype 9 Overview

Adeno-associated virus serotype 9 (AAV9) is a viral vector used for gene therapy, particularly for delivering therapeutic genes to specific tissues. It exhibits broad tropism but is particularly efficient at transducing cardiac muscle, skeletal muscle, and the central nervous system. AAV9 is engineered to carry a therapeutic transgene under the control of tissue- or disease-specific promoters, enabling targeted gene expression.

Mechanism of Action

Delivers therapeutic DNA encoding a corrective transgene via receptor-mediated endocytosis, leading to expression of the therapeutic protein in target cells.

Biological Functions

Gene delivery
Transduction
Gene expression

Disease Associations

Therapeutic agent for genetic diseases
Treatment of neurological disorders
Treatment of cardiac disorders

Safety Considerations

  • Pre-existing immunity to AAV
  • Off-target transduction
  • Limited packaging capacity