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In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations

27 Nov 2022

Abstract excerpt

The tremendous evolution of genome-editing tools in the last two decades has provided innovative and effective approaches for gene therapy of congenital and acquired diseases. Zinc-finger nucleases (ZFNs), transcription activator- like effector nucleases (TALENs) and CRISPR-Cas9 have been already applied by ex vivo hematopoietic stem cell (HSC) gene therapy in genetic diseases (i.e., Hemoglobinopathies, Fanconi...

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PMID: 36553489
DOI: 10.3390/genes13122222

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