Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research
has produced several phase 1-2a trials. Here we provide an overview of the mechanisms …

One of the major goals in the inherited retinal disease (IRD) field is to develop an effective
therapy that can be applied to as many patients as possible. Significant progress has …

Inherited retinal diseases encompass a highly heterogenous group of disorders caused by a
wide range of genetic variants and with diverse clinical symptoms that converge in the …

Inherited retinal dystrophies (IRDs) are a heterogeneous group of diseases that affect more
than 2 million people worldwide. Gene therapy (GT) has emerged as an exciting treatment …

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

Inherited retinal dystrophies [IRDs] are a common cause of severe vision loss resulting from
pathogenic genetic variants. The eye is an attractive target organ for testing clinical …

Gene editing and gene regulatory fields are continuously developing new and safer tools
that move beyond the initial CRISPR/Cas9 technology. As more advanced applications are …

Inherited retinal diseases (IRDs) are vision-threatening retinal disorders caused by
pathogenic variants of genes related to visual functions. Genomic analyses in patients with …

Inherited retinal diseases (IRDs) encompass a diverse group of genetic disorders that lead
to progressive visual impairment and blindness. Over the years, considerable strides have …

The CRISPR/Cas9 system of genome editing has revolutionized molecular biology, offering
a simple, and relatively inexpensive method of creating precise DNA edits. It has potential …