ShH10-CRISPR-Cas9-Aqp1

ShH10-CRISPR-Cas9-Aqp1 is a gene therapy approach that uses an adeno-associated virus (AAV) of the ShH10 serotype to deliver a CRISPR-Cas9 gene editing system specifically targeting the aquaporin 1 (Aqp1) gene in ocular tissues. The therapeutic mechanism disrupts the Aqp1 gene in the ciliary body epithelium, resulting in decreased aqueous humor production and therefore reduced intraocular pressure (IOP). Preclinical studies in mice have demonstrated effective genome editing at the Aqp1 locus, sustained IOP lowering, and preservation of retinal ganglion cells. The therapy is being explored primarily for glaucoma treatment, aiming for a long-term IOP reduction from a single intraocular injection[1][3][5][7].