Nonsense suppression induced readthrough of a novel PAX6 mutation in patient ‐derived cells of congenital aniridia

ConclusionOur data expanded the genetic and phenotypic variations of congenital aniridia, and showed the therapeutic effect of nonsense suppression on this disease using patient ‐derived cells.

Source: Molecular Genetics & Genomic Medicine - March 4, 2020 Category: Genetics & Stem Cells Authors: Xiaoliang Liu, Yuanyuan Zhang, Bijun Zhang, Haiming Gao, Chuang Qiu Tags: ORIGINAL ARTICLE Source Type: research