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Article

Optimized Prime Editing of Human Induced Pluripotent Stem Cells to Efficiently Generate Isogenic Models of Mendelian Diseases.

International journal of molecular sciences - 26 Dec 2024

Cerna-Chavez Rodrigo, Ortega-Gasco Alba, Baig Hafiz Muhammad Azhar, Ehrenreich Nathan, Metais Thibaud, Scandura Michael J, Bujakowska Kinga, Pierce Eric A, Garita-Hernandez Marcela

Abstract excerpt

Prime editing (PE) is a CRISPR-based tool for genome engineering that can be applied to generate human induced pluripotent stem cell (hiPSC)-based disease models. PE technology safely introduces point mutations, small insertions, and deletions (indels) into the genome. It uses a Cas9-nickase (nCas9) fused to a reverse transcriptase (RT) as an editor and a PE guide RNA (pegRNA), which introduces the desired edit...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to RNA, Guide, CRISPR-Cas Systems

Source

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Identifiers

PMID: 39795970
DOI: 10.3390/ijms26010114
PMCID: PMC11719581

Genes

NMNAT1
PRPF3
PRPF8
SSB

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

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