Gene therapy requires a vector: a vehicle used to deliver genetic material into cells. Because viruses do this naturally, they make good vectors. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.

 

Vectors based on adeno-associated virus (AAV) are the current gold standard for human gene therapy.

 

RENOGENYX is engineering all CRISPR components within FSHD-specific therapeutic cassettes for AAV-mediated gene therapy to skeletal muscles.

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