HAL (France)
open access
CRISPR-based strategies for RHO knockout across mouse and retinal organoids
Retinitis pigmentosa is a rare inherited retinal disease that affects 1 in 4,000 people and leads to blindness. Autosomal dominant forms (adRP) account for 20-30% of patients. Mutations in the RHO gene, which encodes rhodopsin in photoreceptors, are the most recurrent in adRP. To directly target the
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