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

CRISPR/Cas9 is a promising technology for gene correction. However, the edition is often biallelic, and uncontrolled small insertions and deletions (indels) concomitant to precise correction are created. Mutation-specific guide RNAs were recently tested to correct dominant inherited diseases, sparin...

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Udgivet i:Stem Cell Reports
Main Authors: Prat, Florence, Toutain, Jérôme, Boutin, Julian, Amintas, Samuel, Cullot, Grégoire, Lalanne, Magalie, Lamrissi-Garcia, Isabelle, Moranvillier, Isabelle, Richard, Emmanuel, Blouin, Jean-Marc, Dabernat, Sandrine, Moreau-Gaudry, François, Bedel, Aurélie
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7486222/
https://ncbi.nlm.nih.gov/pubmed/32795423
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2020.07.015
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