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2016 ; 127
(21
): 2525-35
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A genome editing primer for the hematologist
#MMPMID27053532
Hoban MD
; Bauer DE
Blood
2016[May]; 127
(21
): 2525-35
PMID27053532
show ga
Gene editing enables the site-specific modification of the genome. These
technologies have rapidly advanced such that they have entered common use in
experimental hematology to investigate genetic function. In addition, genome
editing is becoming increasingly plausible as a treatment modality to rectify
genetic blood disorders and improve cellular therapies. Genome modification
typically ensues from site-specific double-strand breaks and may result in a
myriad of outcomes. Even single-strand nicks and targeted biochemical
modifications that do not permanently alter the DNA sequence (epigenome editing)
may be powerful instruments. In this review, we examine the various technologies,
describe their advantages and shortcomings for engendering useful genetic
alterations, and consider future prospects for genome editing to impact
hematology.