Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Summary The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR‐associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genet...

Full description

Saved in:
Bibliographic Details
Published in:British journal of haematology Vol. 185; no. 5; pp. 821 - 835
Main Authors: Jensen, Trine I., Axelgaard, Esben, Bak, Rasmus O.
Format: Journal Article
Language:English
Published: England Blackwell Publishing Ltd 01-06-2019
Subjects:
CAR
Cas9
Chimeric antigen receptors
CRISPR
Disruption
Genetic disorders
Genetic modification
Genomes
haematology
Hematology
Lymphocytes
Lymphocytes T
monogenic
CRISPR
haematology
Cas9
CAR
monogenic
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Summary The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR‐associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genetic therapies that can be applied to haematological disorders. Here we review recent technological advances in the CRISPR/Cas9 methodology and applications in haematology for curing monogenic genetic disorders and for engineering novel chimeric antigen receptor (CAR) T cells to treat haematological malignancies. Furthermore, we discuss current challenges for full clinical implementation of CRISPR/Cas9, and reflect on future trajectories of the technology.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:0007-1048
1365-2141
DOI:10.1111/bjh.15851