Screening of CRISPR/Cas base editors to target the AMD high-risk Y402H complement factor H variant

PURPOSE: To evaluate the efficacy of using a CRISPR/Cas-mediated strategy to correct a common high-risk allele that is associated with age-related macular degeneration (AMD; rs1061170; NM_000186.3:c.1204T>C; NP_000177.2:p.His402Tyr) in the complement factor H (CFH) gene. METHODS: A human embryoni...

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Bibliografski detalji
Izdano u:Mol Vis
Glavni autori: Tran, Minh Thuan Nguyen, Khalid, Mohd Khairul Nizam Mohd, Pébay, Alice, Cook, Anthony L., Liang, Helena H., Wong, Raymond C.B., Craig, Jamie E., Liu, Guei-Sheung, Hung, Sandy S., Hewitt, Alex W.
Format: Artigo
Jezik:Inglês
Izdano: Molecular Vision 2019
Teme:
Research Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6441356/
https://ncbi.nlm.nih.gov/pubmed/30996586
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