Molecular Classification
Viral vector, Adeno-associated virus, Gene therapy vector
Other Names
rAAVrh74, Rh74
Disease Roles
Gene therapy for Duchenne muscular dystrophyGene therapy for Hemophilia B

Adeno-associated Virus Vector Serotype rh74 Overview

Adeno-associated virus serotype rh74 (AAVrh74) is a recombinant viral vector derived from a naturally occurring AAV isolated from rhesus macaques. It is used for gene therapy applications, particularly for neuromuscular diseases like Duchenne muscular dystrophy and hemophilia B, due to its high muscle tropism and low pre-existing immunity in humans. Engineered variants are available to reduce liver tropism.

Mechanism of Action

Delivery of therapeutic gene to target cells

Biological Functions

Gene delivery
Transduction
Gene transfer

Disease Associations

Gene therapy for Duchenne muscular dystrophy
Gene therapy for Hemophilia B

Safety Considerations

  • Immunogenicity
  • Off-target transduction
  • Potential for insertional mutagenesis

Interacting Drugs

BEQVEZ™