CRISPR/Cas9-Based Targeted Genome Editing for Correction of Recessive Dystrophic Epidermolysis Bullosa Using iPS Cells
September 2019
in “
Journal of Investigative Dermatology
”
CRISPR/Cas9 genome editing COL7A1 gene recessive dystrophic epidermolysis bullosa RDEB induced pluripotent stem cells iPSCs exon 19 exon 32 3D skin constructs type VII collagen anchoring fibrils xeno-free iPSC reprogramming GMP certified Cas9 protein chemically modified synthetic guide RNA single-stranded DNA repair templates CRISPR gene editing stem cells skin constructs collagen guide RNA DNA repair templates
Studysummary In this study, researchers used a CRISPR-based method to correct mutations in the COL7A1 gene in stem cells from RDEB patients, restoring normal collagen expression in engineered skin grafts in mice.
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