Efficient Genome Editing Using CRISPR/Cas9 Ribonucleoprotein Approach in iPS Cells for Recessive Dystrophic Epidermolysis Bullosa
April 2019
in “
Journal of Investigative Dermatology
”
CRISPR/Cas9 ribonucleoprotein COL7A1 gene recessive dystrophic epidermolysis bullosa induced pluripotent stem cells homology-directed repair keratinocytes fibroblasts 3D skin constructs type VII collagen anchoring fibrils xeno-free reprogramming GMP certified Cas9 protein chemically modified synthetic guide RNA single-stranded DNA CRISPR RNP iPSCs HDR GMP Cas9 ssDNA
Studysummary This study demonstrated that gene-corrected 3D skin constructs from RDEB patient-derived iPSCs, grafted onto immunocompetent mice, showed normal collagen VII expression after two months.
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