CRISPR/Cas9 genome engineering in hematopoietic cells
Autor: | Duran Sürün, Frank Schnütgen, Harald von Melchner |
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Rok vydání: | 2018 |
Předmět: |
Gene Editing
0301 basic medicine Flexibility (engineering) Acquired Immunodeficiency Syndrome Cas9 Genetic Therapy Computational biology Biology Acquired immune system Hematologic Diseases Genome engineering 03 medical and health sciences Haematopoiesis 030104 developmental biology Genome editing Neoplasms Gene Targeting Drug Discovery Animals Humans Molecular Medicine CRISPR CRISPR-Cas Systems Gene |
Zdroj: | Drug Discovery Today: Technologies. 28:33-39 |
ISSN: | 1740-6749 |
DOI: | 10.1016/j.ddtec.2018.08.001 |
Popis: | 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. |
Databáze: | OpenAIRE |
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