Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

Genome-editing technologies have ushered in a new era in gene therapy, providing novel
therapeutic strategies for a wide range of diseases, including both genetic and nongenetic …

Therapeutic genome editing requires effective and targeted delivery methods. The delivery
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …

Implementation of therapeutic in vivo gene editing using CRISPR/Cas relies on potent
delivery of gene editing tools. Administration of ribonucleoprotein (RNP) complexes …

This review provides comprehensive information about the advances in gene therapy in the
anterior segment of the eye, including cornea, conjunctiva, lacrimal gland, and trabecular …

Retinopathy of prematurity (ROP) is a rare proliferative ocular disorder in preterm infants.
Because of the advancements in neonatal care, the incidence of ROP has increased …

Macroautophagy/autophagy plays an important role in regulating cellular homeostasis and
influences the pathogenesis of degenerative diseases. Tendinopathy is characterized by …

Abstract The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9
system, a groundbreaking innovation in genetic engineering, has revolutionized our …

Genetic medicine is offering hope as new therapies are emerging for many previously
untreatable diseases. The eye is at the forefront of these advances, as exemplified by the …

Many clinical trials using gene therapy have shown significant therapeutic benefits and
exceptional safety records. Increasing evidence is verifying the long sought-after promise …