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CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING

Galaxy Y Cho1,2,3, Kellie A Schaefer4, Alexander G Bassuk5

  • 1Institute of Human Nutrition, Columbia Stem Cell Initiative, College of Physicians and Surgeons, Columbia University, New York, New York.

Retina (Philadelphia, Pa.)
|May 11, 2018
PubMed
Abstract

Insights

Clustered regularly interspaced short palindromic repeats (CRISPR) gene editing shows promise for treating eye diseases. Ongoing research focuses on improving CRISPR specificity and detecting off-target mutations for safe clinical application.

Area of Science:

  • Genetics and Genomics
  • Ophthalmology
  • Molecular Biology

Background:

  • CRISPR technology offers revolutionary potential for genetic disease treatment.
  • Concerns exist regarding unintended genetic alterations (off-target mutations) during CRISPR genome surgery.
  • Ophthalmology is a key area for CRISPR's clinical application due to its potential to repair retinal lesions.

Purpose of the Study:

  • To review methods for detecting CRISPR off-target mutations.
  • To discuss the current and future clinical applications of CRISPR in ophthalmology.
  • To address safety concerns related to CRISPR's clinical use.

Main Methods:

  • Literature review of CRISPR off-targeting detection methods.
  • Analysis of CRISPR's current standing in clinical settings, particularly ophthalmology.
  • Examination of advancements in CRISPR specificity and efficacy.

Main Results:

  • Significant progress has been made in developing methods to detect CRISPR off-target effects.
  • CRISPR tools have seen increased efficacy and specificity in recent years.
  • While a perfect specificity method is not yet available, advancements are promising.

Conclusions:

  • CRISPR gene editing holds immense potential as a therapeutic tool for ophthalmology.
  • Further improvements in specificity and rigorous screening for off-target mutations are crucial for patient application.
  • Optimizing CRISPR technology will maximize its potential for treating retinal diseases.

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