A CRISPR view of hematopoietic stem cells: Moving innovative bioengineering into the clinic

Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome engineering has emerged as a powerful tool to modify precise genomic sequences with unparalleled accuracy and efficiency. Major advances in CRISPR technologies over the last 5 years have fueled the development of novel tec...

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Bibliographic Details
Published in:American journal of hematology 2022-09, Vol.97 (9), p.1226-1235
Main Authors: Worthington, Atesh K., Forsberg, E. Camilla
Format: Article
Language:English
Subjects:
Bioengineering
CRISPR
CRISPR-Cas Systems
Genome editing
Genomics
Genomics - methods
Hematologic Diseases - genetics
Hematologic Diseases - therapy
Hematological diseases
Hematology
Hematopoietic Stem Cells
Humans
Stem cells
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Summary:Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome engineering has emerged as a powerful tool to modify precise genomic sequences with unparalleled accuracy and efficiency. Major advances in CRISPR technologies over the last 5 years have fueled the development of novel techniques in hematopoiesis research to interrogate the complexities of hematopoietic stem cell (HSC) biology. In particular, high throughput CRISPR based screens using various “flavors” of Cas coupled with sequencing and/or functional outputs are becoming increasingly efficient and accessible. In this review, we discuss recent achievements in CRISPR‐mediated genomic engineering and how these new tools have advanced the understanding of HSC heterogeneity and function throughout life. Additionally, we highlight how these techniques can be used to answer previously inaccessible questions and the challenges to implement them. Finally, we focus on their translational potential to both model and treat hematological diseases in the clinic. The advent of CRISPR/Cas genome engineering has pushed the envelope of hematopoiesis research at the bench, improving our understanding of hematopoietic stem cell biology. The advances made have rapidly translated to novel CRISPR/Cas based therapies, improving how hematological diseases are treated in the clinic.
ISSN:0361-8609
1096-8652
DOI:10.1002/ajh.26588