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Target intelligence / Profile preview
Adeno-associated Virus Vector Serotype rh74 (AAVrh74)
- Target
- AAVrh74
- Molecular classification
- Viral vector, Adeno-associated virus, Gene therapy vector
01
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.
- Other names
- rAAVrh74Rh74
02
Mechanism of action
Delivery of therapeutic gene to target cells
03
Biological functions
Gene deliveryTransductionGene transfer
04
Disease associations
Gene therapy for Duchenne muscular dystrophyGene therapy for Hemophilia B
05
Safety considerations
ImmunogenicityOff-target transductionPotential for insertional mutagenesis
06
Interacting drugs
BEQVEZ™
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