AAV-HITI gene editing therapy

AAV-HITI (Adeno-Associated Viral vector-mediated Homology-Independent Targeted Integration) gene editing therapy is a preclinical-stage gene editing platform developed by researchers at the Telethon Institute of Genetics and Medicine (TIGEM). The platform utilizes CRISPR-Cas9 machinery delivered via two co-administered adeno-associated virus serotype 8 (AAV8) vectors to achieve stable, promoterless transgene knock-in. Unlike traditional homology-directed repair (HDR), which is often inefficient in non-dividing cells, HITI leverages the non-homologous end joining (NHEJ) DNA repair pathway. This allows for efficient gene insertion in both dividing and non-dividing cells, such as hepatocytes and photoreceptors. The therapy has demonstrated therapeutic benefit in mouse models of inherited diseases, including hemophilia A, lysosomal storage diseases, and autosomal dominant retinitis pigmentosa (ADRP), by targeting genomic loci such as albumin or rhodopsin (RHO).

Other names

Adeno-Associated Viral vector-mediated Homology-Independent Targeted Integration gene editing therapy