Open access
Aug 2026
Generation of isogenic gene-corrected cell lines from a USH2A-RP patient-derived iPS cell line.
Comparative studies using induced pluripotent stem cells (iPSCs) from patients with those from healthy individuals as controls are flawed by genetic background contribution to disease phenotype. Here, we used precise gene editing to generate gene-corrected isogenic control lines for a single pathogenic variant in the U...
Verónica del Buey Furió, Belén García-Bohórquez, G. García-García et al.
· Stem Cell Research · 0 citations