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2017 ; 19
(7
): 32
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Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing
#MMPMID28550381
Chadwick AC
; Musunuru K
Curr Atheroscler Rep
2017[Jul]; 19
(7
): 32
PMID28550381
show ga
PURPOSE OF REVIEW: Clustered regularly interspaced short palindromic repeat
(CRISPR)/CRISPR-associated 9 (Cas9) has recently emerged as a top genome editing
technology and has afforded investigators the ability to more easily study a
number of diseases. This review discusses CRISPR/Cas9's advantages and
limitations and highlights a few recent reports on genome editing applications
for alleviating dyslipidemia through disruption of proprotein convertase
subtilisin/kexin type 9 (PCSK9). RECENT FINDINGS: Targeting of mouse Pcsk9 using
CRISPR/Cas9 technology has yielded promising results for lowering total
cholesterol levels, and several recent findings are highlighted in this review.
Reported on-target mutagenesis efficiency is as high as 90% with a subsequent 40%
reduction of blood cholesterol levels in mice, highlighting the potential for use
as a therapeutic in human patients. The ability to characterize and treat
diseases is becoming easier with the recent advances in genome editing
technologies. In this review, we discuss how genome editing strategies can be of
use for potential therapeutic applications.
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free
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