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AAV-mediated human CNGB3 restores cone function in an all-cone mouse model of CNGB3 achromatopsia
Complete congenital achromatopsia is a devastating hereditary visual disorder. Mutations in the CNGB3 gene account for more than 50% of all known cases of achromatopsia. This work investigated the efficiency of subretinal (SR) delivered AAV8 (Y447, 733F) vector containing a human PR2.1 promoter and...
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| Pubblicato in: | J Biomed Res |
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| Autori principali: | , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Editorial Department of Journal of Biomedical Research
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7183301/ https://ncbi.nlm.nih.gov/pubmed/32305965 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.7555/JBR.33.20190056 |
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