Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9
Jiahui Wu, Oliver H. Bell, David A. Copland, Alison Young, John R. Pooley, Ryea Maswood, Rachel S Evans, Peng T. Khaw, Robin R. Ali, Andrew D. Dick, Colin J. Chu
Abstract
; p < 0.01). ShH10 could transduce human ciliary body from post-mortem donor eyes in ex vivo culture with indel formation detectable in the Aquaporin 1 locus. Clinical translation of this approach to patients with glaucoma may permit long-term reduction of IOP following a single injection.
Topics & Concepts
CRISPRGlaucomaGenetic enhancementCiliary bodyBiologyCas9GeneGenome editingCell biologyOphthalmologyGeneticsMedicineNeuroscienceRetinal Development and DisordersCRISPR and Genetic EngineeringRetinopathy of Prematurity Studies