AAV-G6PC genome editing therapy

**AAV-G6PC genome editing therapy** is an academic, preclinical liver-directed **CRISPR-based gene-editing approach** for glycogen storage disease type Ia. It uses two adeno-associated virus vectors: a CRISPR vector encoding *Streptococcus pyogenes* Cas9 and a donor vector carrying a guide RNA plus a therapeutic *G6PC* transgene. Cas9-mediated cleavage at the endogenous *G6PC* locus enables homology-directed chromosomal insertion of the donor transgene, aiming to restore durable hepatocyte expression of glucose-6-phosphatase-alpha and avoid the progressive loss of episomal AAV genomes during liver growth. The approach has produced metabolic correction in murine and canine GSD Ia models but has not been identified as a named clinical product or an active human clinical program. ([insight.jci.org](https://insight.jci.org/articles/view/181760))

Other names

AAV-G6PC genome editing therapyAAV-G-6PC genome editing therapyAAV-G 6PC genome editing therapy