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Technical considerations for the use of CRISPR/Cas9 in hematology research

Gundry, Michael C.; Dever, Daniel P.; Yudovich, David LU ; Bauer, Daniel E.; Haas, Simon; Wilkinson, Adam C. and Singbrant, Sofie LU (2017) In Experimental Hematology
Abstract

The hematopoietic system is responsible for transporting oxygen and nutrients, fighting infections, and repairing tissue damage. Hematopoietic system dysfunction therefore causes a range of serious health consequences. Lifelong hematopoiesis is maintained by repopulating multipotent hematopoietic stem cells (HSCs) that replenish shorter-lived, mature blood cell types. A prokaryotic mechanism of immunity, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system, has been recently "repurposed" to mutate mammalian genomes efficiently and in a sequence-specific manner. The application of this genome-editing technology to hematology has afforded new approaches for functional genomics and even the prospect... (More)

The hematopoietic system is responsible for transporting oxygen and nutrients, fighting infections, and repairing tissue damage. Hematopoietic system dysfunction therefore causes a range of serious health consequences. Lifelong hematopoiesis is maintained by repopulating multipotent hematopoietic stem cells (HSCs) that replenish shorter-lived, mature blood cell types. A prokaryotic mechanism of immunity, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system, has been recently "repurposed" to mutate mammalian genomes efficiently and in a sequence-specific manner. The application of this genome-editing technology to hematology has afforded new approaches for functional genomics and even the prospect of "correcting" dysfunctional HSCs in the treatment of serious genetic hematological diseases. In this Perspective, we provide an overview of three recent CRISPR/Cas9 methods in hematology: gene disruption, gene targeting, and saturating mutagenesis. We also summarize the technical considerations and advice provided during the May 2017 International Society of Experimental Hematology New Investigator Committee webinar on the same topic.

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publication status
published
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Experimental Hematology
publisher
Elsevier
external identifiers
  • scopus:85028735553
  • wos:000411778100002
ISSN
0301-472X
DOI
10.1016/j.exphem.2017.07.006
language
English
LU publication?
yes
id
83067fb5-1c08-4ef3-b29c-7a551c00b832
date added to LUP
2017-10-10 15:12:17
date last changed
2018-10-03 11:03:40
@article{83067fb5-1c08-4ef3-b29c-7a551c00b832,
  abstract     = {<p>The hematopoietic system is responsible for transporting oxygen and nutrients, fighting infections, and repairing tissue damage. Hematopoietic system dysfunction therefore causes a range of serious health consequences. Lifelong hematopoiesis is maintained by repopulating multipotent hematopoietic stem cells (HSCs) that replenish shorter-lived, mature blood cell types. A prokaryotic mechanism of immunity, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system, has been recently "repurposed" to mutate mammalian genomes efficiently and in a sequence-specific manner. The application of this genome-editing technology to hematology has afforded new approaches for functional genomics and even the prospect of "correcting" dysfunctional HSCs in the treatment of serious genetic hematological diseases. In this Perspective, we provide an overview of three recent CRISPR/Cas9 methods in hematology: gene disruption, gene targeting, and saturating mutagenesis. We also summarize the technical considerations and advice provided during the May 2017 International Society of Experimental Hematology New Investigator Committee webinar on the same topic.</p>},
  author       = {Gundry, Michael C. and Dever, Daniel P. and Yudovich, David and Bauer, Daniel E. and Haas, Simon and Wilkinson, Adam C. and Singbrant, Sofie},
  issn         = {0301-472X},
  language     = {eng},
  publisher    = {Elsevier},
  series       = {Experimental Hematology},
  title        = {Technical considerations for the use of CRISPR/Cas9 in hematology research},
  url          = {http://dx.doi.org/10.1016/j.exphem.2017.07.006},
  year         = {2017},
}