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

27.05.2016 | Progress in Hematology

Gene editing and its application for hematological diseases

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

Erschienen in: International Journal of Hematology | Ausgabe 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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Metadaten
Titel
Gene editing and its application for hematological diseases
verfasst von
Mark J. Osborn
Joseph J. Belanto
Jakub Tolar
Daniel F. Voytas
Publikationsdatum
27.05.2016
Verlag
Springer Japan
Erschienen in
International Journal of Hematology / Ausgabe 1/2016
Print ISSN: 0925-5710
Elektronische ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-016-2017-z

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