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International Journal of Hematology
Published in: International Journal of Hematology 1/2016

01-07-2016 | Progress in Hematology

Gene editing and its application for hematological diseases

Authors: Mark J. Osborn, Joseph J. Belanto, Jakub Tolar, Daniel F. Voytas

Published in: International Journal of Hematology | Issue 1/2016

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Abstract

The use of precise, rationally designed gene-editing nucleases allows for targeted genome and transcriptome modification, and at present, four major classes of nucleases are being employed: zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), meganucleases (MNs), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9. Each reagent shares the ability to recognize and bind a target sequence of DNA. Depending on the properties of the reagent, the DNA can be cleaved on one or both strands, or epigenetic changes can be mediated. These novel properties can impact hematological disease by allowing for: (1) direct modification of hematopoietic stem/progenitor cells (HSPCs), (2) gene alteration of hematopoietic lineage committed terminal effectors, (3) genome engineering in non-hematopoietic cells with reprogramming to a hematopoietic phenotype, and (4) transcriptome modulation for gene regulation, modeling, and discovery.
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Metadata
Title
Gene editing and its application for hematological diseases
Authors
Mark J. Osborn
Joseph J. Belanto
Jakub Tolar
Daniel F. Voytas
Publication date
01-07-2016
Publisher
Springer Japan
Published in
International Journal of Hematology / Issue 1/2016
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-016-2017-z

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