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 embry...

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Bibliographic Details
Main Authors: Minh Thuan Nguyen Tran, Mohd Khairul Nizam Mohd Khalid, Alice Pébay, Anthony L. Cook, Helena H. Liang, Raymond C.B. Wong, Jamie E. Craig, Guei-Sheung Liu, Sandy S. Hung, Alex W. Hewitt
Format: Article
Language:English
Published: Molecular Vision 2019-03-01
Series:Molecular Vision
Subjects:
Online Access:http://www.molvis.org/molvis/v25/174/