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Updated: Mar 30, 2026

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
Published on: September 25, 2019
[CRISPR-Cas9, a new chance for somatic gene therapy]
1UMR 7268 ADÉS, Aix-Marseille, Université/EFS/CNRS, Espace éthique méditerranéen, hôpital d'adultes la Timone, 264, rue Saint-Pierre, 13385 Marseille Cedex 05, France ; CoReBio PACA, case 901, parc scientifique de Luminy, 13288 Marseille Cedex 09, France.
Abstract:
Targeted modification of genes ("gene editing") is made much easier by the recently developed CRISPR-Cas9 system. This has raised alarm about possible uses of this technology for germline modification of the human genome; however this technology has less controversial applications, notably for somatic gene therapy with already some striking demonstrations in animal systems. Because of its precision and relative ease of use, CRISPR can be expected to drive a revolution in gene therapy and to turn it into a more mainstream approach.
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