Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Trine I Jensen; Esben Axelgaard; Rasmus O Bak

doi:10.1111/bjh.15851

Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Br J Haematol. 2019 Jun;185(5):821-835. doi: 10.1111/bjh.15851. Epub 2019 Mar 12.

Authors

Trine I Jensen¹, Esben Axelgaard¹, Rasmus O Bak^{1

2}

Affiliations

¹ Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
² Aarhus Institute of Advanced Studies (AIAS), Aarhus University, Aarhus C, Denmark.

PMID: 30864164
DOI: 10.1111/bjh.15851

Abstract

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.

Keywords: CAR; CRISPR; Cas9; haematology; monogenic.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

CRISPR-Cas Systems / genetics*
Gene Editing / methods*
Hematologic Diseases / genetics*
Humans