Since long before the first approval of gene therapy for retinal disease, ocular gene therapy
has captured the hopes of patients, clinicians, and scientists alike. Indeed, the retina …

Gene therapy is a flourishing field with the potential to revolutionize the treatment of genetic
diseases. The emergence of CRISPR-Cas9 has significantly advanced targeted and efficient …

CRISPR-Cas-based genome editing technologies could, in principle, be used to treat a wide
variety of inherited diseases, including genetic disorders of vision. Programmable CRISPR …

Age-related macular degeneration (AMD) is a complex and multifactorial disease and a
leading cause of irreversible blindness in the elderly population. The anti-vascular …

Neurological diseases associated with pathogenic variants in a specific gene, or even with a
specific pathogenic variant, can show profound phenotypic variation with regard to symptom …

Autosomal dominant disorders present unique challenges, as therapeutics must often
distinguish between healthy and diseased alleles while maintaining high efficiency …

Mutations in the Crumbs homolog 1 (CRB1) gene lead to severe inherited retinal
dystrophies (IRDs), accounting for nearly 80,000 cases worldwide. To date, there is no …

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of
diseases that are one of the leading causes of vision loss in young and aged individuals …

With a common aim of restoring physiological function of defective cells, optogenetics and
targeted gene therapies have shown great clinical potential and novelty in the branch of …

The mouse and human retina contain three major Crumbs homologue-1 (CRB1) isoforms.
CRB1-A and CRB1-B have cell-type-specific expression patterns making the choice of gene …