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Abstract Retinitis pigmentosa (RP), a leading cause of inherited blindness, results in a subset of cases from phosphodiesterase 6 (PDE6) deficiency. We evaluated a novel gene therapy construct, AAV8-RK-PDE6B-3′UTR, in the rd10 mouse model which has a homozygous missense variant that disrupts the function of the β-subunit of PDE6.
As a journalist, you can create a free Muck Rack account to customize your profile, list your contact preferences, and upload a portfolio of your best work.
