Mutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells

Published 2020 View Full Article

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Title
Mutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells
Authors
Keywords
CRISPR/Cas9, gene therapy, iPSCs, precise genome editing, congenital erythropoietic porphyria, compound heterozygous recessive disease, mutation specific
Journal
Stem Cell Reports
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-08-13
DOI
10.1016/j.stemcr.2020.07.015

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