R-02 gRNA

R-02 gRNA is a specific guide RNA component of EBT-101, an investigational multiplex CRISPR-Cas9 gene therapy developed by Excision BioTherapeutics for the treatment of HIV-1. EBT-101 utilizes an adeno-associated virus (AAV9) vector to deliver the Cas9 nuclease along with multiple guide RNAs, including R-02, which are designed to target highly conserved regions of the integrated HIV-1 proviral DNA. Specifically, R-02 gRNA directs the Cas9 enzyme to the Gag region of the viral genome. By inducing double-strand breaks at multiple sites (in combination with other gRNAs targeting the LTR regions), the therapy aims to excise large segments of the HIV-1 DNA, thereby preventing viral replication and potentially providing a functional cure for individuals living with HIV.

Other names

R-02 guide RNAR02 guide RNAR 02 guide RNAGuide RNA 2