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2017 ; 28
(3
): 252-259
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CRISPR applications in ophthalmologic genome surgery
#MMPMID28141764
Cabral T
; DiCarlo JE
; Justus S
; Sengillo JD
; Xu Y
; Tsang SH
Curr Opin Ophthalmol
2017[May]; 28
(3
): 252-259
PMID28141764
show ga
PURPOSE OF REVIEW: The present review seeks to summarize and discuss the
application of clustered regularly interspaced short palindromic repeats
(CRISPR)-associated systems (Cas) for genome editing, also called genome surgery,
in the field of ophthalmology. RECENT FINDINGS: Precision medicine is an emerging
approach for disease treatment and prevention that takes into account the
variability of an individual's genetic sequence. Various groups have used
CRISPR-Cas genome editing to make significant progress in mammalian preclinical
models of eye disease, the basic science of eye development in zebrafish, the in
vivo modification of ocular tissue, and the correction of stem cells with
therapeutic applications. In addition, investigators have creatively used the
targeted mutagenic potential of CRISPR-Cas systems to target pathogenic alleles
in vitro. SUMMARY: Over the past year, CRISPR-Cas genome editing has been used to
correct pathogenic mutations in vivo and in transplantable stem cells. Although
off-target mutagenesis remains a concern, improvement in CRISPR-Cas technology
and careful screening for undesired mutations will likely lead to clinical eye
therapeutics employing CRISPR-Cas systems in the near future.
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