Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation

In the field of hematology, gene therapies based on integrating vectors have reached outstanding results for a number of human diseases. With the advent of novel programmable nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addit...

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Bibliographic Details
Published in:Frontiers in genome editing 2021-03, Vol.3, p.618378-618378
Main Authors: Ferrari, Samuele, Vavassori, Valentina, Canarutto, Daniele, Jacob, Aurelien, Castiello, Maria Carmina, Javed, Attya Omer, Genovese, Pietro
Format: Article
Language:English
Subjects:
CRISPR/Cas
gene editing
gene therapy
Genome Editing
hematological diseases
hematopoietic stem cell
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Summary:In the field of hematology, gene therapies based on integrating vectors have reached outstanding results for a number of human diseases. With the advent of novel programmable nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addition to site-specific modification of the genome, holding the promise for safer genetic manipulation. Here we review the state of the art of ex vivo gene editing with programmable nucleases in human hematopoietic stem and progenitor cells (HSPCs). We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
ISSN:2673-3439
2673-3439
DOI:10.3389/fgeed.2021.618378