PMID- 28141764
DCOM- 20170418
LR  - 20180502
IS  - 1531-7021 (Electronic)
IS  - 1040-8738 (Linking)
VI  - 28
IP  - 3
DP  - 2017 May
TI  - CRISPR applications in ophthalmologic genome surgery.
PG  - 252-259
LID - 10.1097/ICU.0000000000000359 [doi]
AB  - 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.
FAU - Cabral, Thiago
AU  - Cabral T
AD  - aJonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory,
      Department of Ophthalmology, Columbia University Medical Center bEdward S
      Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA
      cDepartment of Ophthalmology, Federal University of Espirito Santo, Vitoria,
      Brazil dDepartment of Ophthalmology, Federal University of Sao Paulo, Sao Paulo, 
      Brazil eState University of New York Downstate Medical Center, Brooklyn, NY, USA 
      fDepartment of Ophthalmology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong
      University, Shanghai, China gDepartment of Pathology & Cell Biology, Institute of
      Human Nutrition, College of Physicians and Surgeons, Columbia University, New
      York, NY, USA.
FAU - DiCarlo, James E
AU  - DiCarlo JE
FAU - Justus, Sally
AU  - Justus S
FAU - Sengillo, Jesse D
AU  - Sengillo JD
FAU - Xu, Yu
AU  - Xu Y
FAU - Tsang, Stephen H
AU  - Tsang SH
LA  - eng
GR  - R21 AG050437/AG/NIA NIH HHS/United States
GR  - R01 EY018213/EY/NEI NIH HHS/United States
GR  - R01 EY026682/EY/NEI NIH HHS/United States
GR  - R01 EY024698/EY/NEI NIH HHS/United States
GR  - P30 EY019007/EY/NEI NIH HHS/United States
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Curr Opin Ophthalmol
JT  - Current opinion in ophthalmology
JID - 9011108
SB  - IM
MH  - *CRISPR-Cas Systems
MH  - Dependovirus/genetics
MH  - Embryonic Stem Cells/transplantation
MH  - Eye Diseases/*genetics
MH  - *Gene Editing
MH  - Genetic Therapy
MH  - Humans
MH  - Induced Pluripotent Stem Cells/transplantation
MH  - Precision Medicine
PMC - PMC5511789
MID - NIHMS873748
EDAT- 2017/02/01 06:00
MHDA- 2017/04/19 06:00
CRDT- 2017/02/01 06:00
PHST- 2017/02/01 06:00 [pubmed]
PHST- 2017/04/19 06:00 [medline]
PHST- 2017/02/01 06:00 [entrez]
AID - 10.1097/ICU.0000000000000359 [doi]
PST - ppublish
SO  - Curr Opin Ophthalmol. 2017 May;28(3):252-259. doi: 10.1097/ICU.0000000000000359.