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Updated: Dec 1, 2025

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
Published on: February 2, 2016
A Genome Editing System for Therapeutical Targeting of Stem Cells
Giacomo Frati1, Annarita Miccio2
1Imagine Institute, Paris, France. giacomo.frati@institutimagine.org.
Abstract:
The CRISPR/Cas9 system can be exploited to disrupt genes or cis-regulatory elements in the genome of human hematopoietic stem cells. Here, we describe a protocol to deliver the CRISPR/Cas9 ribonucleoprotein complexes into primary human hematopoietic stem cells and to evaluate the engraftment and multilineage differentiation of edited cells in immunodeficient mice. This procedure allows the editing of a high proportion of long-term repopulating hematopoietic stem cells.
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