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Fidelle Chahine Karam on Muck Rack

Fidelle Chahine Karam

As seen in: Nature
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AAV8- PDE6B with 3’UTR provides rescue of photoreceptor structure and function in the rd10 mouse model of retinitis pigmentosa

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By Anson Cheng, Leszek Lisowski, John Grigg, Ian Alexander, Robyn Jamieson, Steven S. Eamegdool, Amin Sabri, Fidelle Chahine Karam
| Nature Verified
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.
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