CRISPR/Cas9 genome engineering in hematopoietic cells

Autor: Duran Sürün, Frank Schnütgen, Harald von Melchner
Rok vydání: 2018
Předmět:
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