The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-
associated nuclease (Cas) system is an adaptive immune defence system that has gradually …

Inherited retinal degenerations (IRDs) are a leading cause of blindness. Although gene-
supplementation therapies have been developed, they are only available for a small …

Gene augmentation and genome editing are promising strategies for the treatment of
monogenic inherited retinal diseases. Although gene augmentation treatments are …

Genome editing provides a new therapeutic strategy to cure genetic diseases. The recently
developed CRISPR-Cas9 base editing technology has shown great potential to repair the …

Novel gene therapy treatments for inherited retinal diseases have been at the forefront of
translational medicine over the past couple of decades. Since the discovery of CRISPR …

The RNA-guided endonuclease system CRISPR-Cas9 has been extensively modified since
its discovery, allowing its capabilities to extend far beyond double-stranded cleavage to high …

Age-related macular degeneration (AMD) is the most common cause of irreversible vision
loss in people over 50 years old in developed countries. Currently, we still lack a …

Background Precision genome engineering, with targeted therapy towards patient-specific
mutations is predicted to be the future of personalized medicine. Ophthalmology is in the …

Human pluripotent stem cells (hPSCs), which include induced pluripotent stem cells and
embryonic stem cells, are powerful tools for studying human development, physiology and …

Genetic diseases are a leading cause of death and disability worldwide with immense
economic and societal burdens. However, currently fewer than 5% of genetic diseases have …