CRISPR/Cas9 genome engineering in hematopoietic cells

Publication date: Available online 25 August 2018Source: Drug Discovery Today: TechnologiesAuthor(s): Duran Sürün, Harald von Melchner, Frank SchnütgenThe 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.
Source: Drug Discovery Today: Technologies - Category: Drugs & Pharmacology Source Type: research