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Stanford researchers have developed a genome engineering platform for generating genetically precise human cellular models of Lowe syndrome, a rare inherited disorder frequently associated with severe ocular complications, including early-onset glaucoma.

Stanford researchers have developed a next-generation programmable transcriptional activation platform, TIGRa, that addresses key limitations of CRISPRa technologies, including large size, limited multiplexing capacity, and delivery constraints.

Stanford scientists have discovered that novel gene therapy and metabolite supplementation approaches show promise in treating ciliopathies.