Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Esben Axelgaard; Trine Ilsø Jensen; Rasmus O. Bak

doi:10.1111/bjh.15851

Therapeutic gene editing in haematological disorders with CRISPR/Cas9

Esben Axelgaard
, Trine Ilsø Jensen
, Rasmus O. Bak

Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avis › Review › Forskning › peer review

39 Citationer (Scopus)

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.

Originalsprog	Engelsk
Tidsskrift	British Journal of Haematology
Vol/bind	185
Nummer	5
Sider (fra-til)	821-835
Antal sider	15
ISSN	0007-1048
DOI	https://doi.org/10.1111/bjh.15851
Status	Udgivet - 2019

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title = "Therapeutic gene editing in haematological disorders with CRISPR/Cas9",

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, CRISPR-CAS9, Cas9, DNA, GENOME, IMMUNODEFICIENCY, INTERLEUKIN-15, MUTATION, REPOPULATING HEMATOPOIETIC STEM, RNA, T-CELLS, TARGET, haematology, monogenic",

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year = "2019",

doi = "10.1111/bjh.15851",

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pages = "821--835",

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T1 - Therapeutic gene editing in haematological disorders with CRISPR/Cas9

AU - Axelgaard, Esben

AU - Jensen, Trine Ilsø

AU - Bak, Rasmus O.

PY - 2019

Y1 - 2019

N2 - 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.

AB - 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.

KW - CAR

KW - CRISPR

KW - CRISPR-CAS9

KW - Cas9

KW - DNA

KW - GENOME

KW - IMMUNODEFICIENCY

KW - INTERLEUKIN-15

KW - MUTATION

KW - REPOPULATING HEMATOPOIETIC STEM

KW - RNA

KW - T-CELLS

KW - TARGET

KW - haematology

KW - monogenic

U2 - 10.1111/bjh.15851

DO - 10.1111/bjh.15851

M3 - Review

C2 - 30864164

SN - 0007-1048

VL - 185

SP - 821

EP - 835

JO - British Journal of Haematology

JF - British Journal of Haematology

IS - 5

ER -

Therapeutic gene editing in haematological disorders with CRISPR/Cas9

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