Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases

Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel g...

Full description

Saved in:
Bibliographic Details
Published in:The FEBS journal 2023-11, Vol.290 (22), p.5248-5269
Main Authors: Sundaresan, Yogapriya, Yacoub, Sam, Kodati, Bindu, Amankwa, Charles E, Raola, Akash, Zode, Gulab
Format: Article
Language:English
Subjects:
CRISPR
CRISPR-Cas Systems - genetics
Editing
Environmental factors
Eye diseases
Gene Editing - methods
Genetic diversity
Genetic modification
Genetic Therapy - methods
Genetic variance
Genome
Genomics
Health services
Immune privilege
Nucleic acids
Phenotypes
Signs and symptoms
Therapeutic applications
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel genetic therapies. Advances in genome editing have revolutionized the field of biomedical science, enabling researchers to understand the biology behind disease mechanisms and allow the treatment of several health conditions, including ocular pathologies. The advent of clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing facilitates efficient and specific genetic modifications in the nucleic acid sequence, resulting in permanent changes at the genomic level. This approach has advantages over other treatment strategies and is promising for the treatment of various genetic and non-genetic ocular conditions. This review provides an overview of the CRISPR/CRISPR-associated protein 9 (Cas9) system and summarizes recent advances in the therapeutic application of CRISPR/Cas9 for the treatment of various ocular pathologies, as well as future challenges.
ISSN:1742-464X
1742-4658
1742-4658
DOI:10.1111/febs.16771