CRISPR/Cas9 genome engineering in hematopoietic cells

  • The development of genome editing tools capable of modifying specific genomic sequences with unprecedented accuracy has opened up a wide range of new possibilities in targeted gene manipulation. In particular, the CRISPR/Cas9 system, a repurposed prokaryotic adaptive immune system, has been widely adopted because of its unmatched simplicity and flexibility. In this review we discuss achievements and current limitations of CRISPR/Cas9 genome editing in hematopoietic cells with special emphasis on its potential use in ex vivo gene therapy of monogenic blood disorders, HIV and cancer.

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Author:Duran Sürün, Harald von Melchner, Frank SchnütgenORCiDGND
URN:urn:nbn:de:hebis:30:3-514012
DOI:https://doi.org/10.1016/j.ddtec.2018.08.001
ISSN:1740-6749
Pubmed Id:https://pubmed.ncbi.nlm.nih.gov/30205879
Parent Title (English):Drug discovery today. Technologies
Publisher:Elsevier
Place of publication:Amsterdam [u.a.]
Contributor(s):Milena Bellin
Document Type:Article
Language:English
Year of Completion:2018
Date of first Publication:2018/08/25
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2019/10/21
Volume:28
Page Number:7
First Page:33
Last Page:39
Note:
© 2018 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
HeBIS-PPN:455688702
Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Licence (German):License LogoCreative Commons - Namensnennung 4.0