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Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

6 Apr 2018

Abstract excerpt

BACKGROUND: Replacement therapy for hemophilia remains a lifelong treatment. Only gene therapy can cure hemophilia at a fundamental level. The clustered regularly interspaced short palindromic repeats-CRISPR associated nuclease 9 (CRISPR-Cas9) system is a versatile and convenient genome editing tool which can be applied to gene therapy for hemophilia. METHODS: A patient's induced pluripotent stem cells (iPSCs)...

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CAR-T cell therapy research
CRISPR and Genetic Engineering
Hemophilia Treatment and Research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 29625575
DOI: 10.1186/s13287-018-0839-8

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