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AAV8-PDE6B with 3’UTR provides durable rescue of photoreceptor structure and function in rd10 mouse model of retinitis pigmentosa

2026-05-20

Abstract excerpt

<title>Abstract</title> <p> Retinitis pigmentosa (RP), a leading cause of inherited blindness, often results from phosphodiesterase 6 (PDE6) deficiency. We evaluated a novel gene therapy construct, AAV8-RK-PDE6B-3’UTR, in the <italic>rd10</italic> mouse model which lacks the functional β-subunit of PDE6. The construct utilises a rhodopsin-kinase promoter, human PDE6B cDNA, and a PDE6B 3’ untranslated region (3...

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Literature Corpus work
674950ed-2c4b-5a84-ae3f-4e5e72ad905c
DOI
10.21203/rs.3.rs-9298568/v1
Open publication

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AAV8-PDE6B with 3’UTR provides durable rescue of photoreceptor structure and function in rd10 mouse model of retinitis pigmentosaDOI 10.21203/rs.3.rs-9298568/v1
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