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

Stem cell reports - 8 Sept 2020

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

Abstract excerpt

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, sparing the wild-type allele. We tested an original approach to correct compound heterozygous recessive mutations. We...

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Identifiers

PMID: 32795423
DOI: 10.1016/j.stemcr.2020.07.015
PMCID: PMC7486222

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UROS

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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