title: Gene editing and its application for hematological diseases.
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.
authors.affiliation: University of Minnesota Twin Cities, Minneapolis, MN, USA.
authors.forename: Mark J
authors.initials: MJ
authors.lastname: Osborn
authors.affiliation: University of Minnesota Twin Cities, Minneapolis, MN, USA.
authors.forename: Joseph J
authors.initials: JJ
authors.lastname: Belanto
authors.affiliation: University of Minnesota Twin Cities, Minneapolis, MN, USA. tolar003@umn.edu.
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.affiliation: University of Minnesota Twin Cities, Minneapolis, MN, USA.
authors.forename: Daniel F
authors.initials: DF
authors.lastname: Voytas
mesh_terms: D004720:Endonucleases / Q000235:genetics; D000072669:Gene Editing / Q000379:methods*; D018390:Gene Targeting / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D006402:Hematologic Diseases / Q000235:genetics / Q000628:therapy*; D006801:Humans
chemical_list: D004720:Endonucleases
keywords: Blood disorders; CRISPR/Cas9; Hematopoiesis; Hematopoietic stem cells; TALEN; ZFN