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Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress.

Biomolecules - 25 Jul 2024

Liu Yuchen, Zong Xin, Cao Wenye, Zhang Wenxi, Zhang Ningzhi, Yang Ning

Abstract excerpt

Retinitis pigmentosa (RP) poses a significant threat to eye health worldwide, with prevalence rates of 1 in 5000 worldwide. This genetically diverse retinopathy is characterized by the loss of photoreceptor cells and atrophy of the retinal pigment epithelium. Despite the involvement of more than 3000 mutations across approximately 90 genes in its onset, finding an effective treatment has been challenging for a...

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Eye Proteins
Gene Editing
Genetic Therapy
Genetic Vectors
Mutation
Retinitis Pigmentosa

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PMID: 39199291
DOI: 10.3390/biom14080903
PMCID: PMC11352491

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