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Article

All-in-one AAV-mediated Nrl gene inactivation rescues retinal degeneration in Pde6a mice.

JCI insight - 20 Dec 2024

Liu Zhiquan, Chen Siyu, Lo Chien-Hui, Wang Qing, Sun Yang

Abstract excerpt

Retinitis pigmentosa (RP) is a complex group of inherited retinal diseases characterized by progressive death of photoreceptor cells and eventual blindness. Pde6a, which encodes a cGMP-specific phosphodiesterase, is a crucial pathogenic gene for autosomal recessive RP (RP43); there is no effective therapy for this form of RP. The compact CRISPR/Staphylococcus aureus Cas9 (CRISPR/SaCas9) system, which can be...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39499900
DOI: 10.1172/jci.insight.178159
PMCID: PMC11665581

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