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 a...

Full description

Saved in:
Bibliographic Details
Published in:Drug discovery today. Technologies 2018-08, Vol.28, p.33-39
Main Authors: Sürün, Duran, von Melchner, Harald, Schnütgen, Frank
Format: Article
Language:English
Subjects:
Acquired Immunodeficiency Syndrome - genetics
Acquired Immunodeficiency Syndrome - therapy
Animals
CRISPR-Cas Systems
Gene Editing
Gene Targeting - methods
Genetic Therapy - methods
Hematologic Diseases - genetics
Hematologic Diseases - therapy
Humans
Neoplasms - genetics
Neoplasms - therapy
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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.
ISSN:1740-6749
1740-6749
DOI:10.1016/j.ddtec.2018.08.001