Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

The eye presents extensive perspectives and challenges for drug delivery, mainly because
of the extraordinary capacity, intrinsic to this path, for drugs to permeate into the main …

Background CEP290-associated inherited retinal degeneration causes severe early-onset
vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly …

In 2001, the first large animal was successfully treated with a gene therapy that restored its
vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness …

Aerobic glycolysis accounts for∼ 80%–90% of glucose used by adult photoreceptors (PRs);
yet, the importance of aerobic glycolysis for PR function or survival remains unclear. Here …

The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65
mutation-associated inherited retinal dystrophy with viable retinal cells represents an …

In vivo genome editing represents an emerging field in the treatment of monogenic
disorders, as it may constitute a solution to the current hurdles in classic gene addition …

Voretigene neparvovec‐rzyl was recently approved for the treatment of Leber congenital
amaurosis, and the use of gene therapy for eye disease is attracting even greater interest …

Diabetic retinopathy (DR) is a vision-impairing complication of diabetes, damaging the
retinal microcirculatory system. Overexpression of VEGF (vascular endothelial growth factor) …

Parkinson's disease, diabetic retinopathy, hyperoxia induced retinopathy, and neuronal
damage resulting from ischemia are among the notable neurodegenerative diseases in …