Gene therapy is a technique involving the modification of an individual's genes for treating a
particular disease. The key to effective gene therapy is an efficient carrier delivery system …

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 …

Retinitis Pigmentosa - StatPearls - NCBI Bookshelf US flag An official website of the United
States government Here's how you know NIH NLM Logo Access keys NCBI Homepage …

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

Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of
diseases and a leading cause of blindness in the working-age population. Even though …

Stargardt disease, one of the most common forms of inherited retinal diseases, affects
individuals worldwide. The primary cause is mutations in the ABCA4 gene, leading to the …

Gene therapies, despite of being a relatively new therapeutic approach, have a potential to
become an important alternative to current treatment strategies in glaucoma. Since …

The rapid evolution of two technologies has greatly transformed the basic, translational, and
clinical research in the mammalian retina. One is the retinal organoid (RO) technology …

Interest in gene therapy medicines is intensifying as the first wave of gene-correcting drugs
is now reaching patient populations. However, efficacy and safety concerns, laborious …

Fibrotic diseases are characterised by myofibroblast differentiation, uncontrolled
pathological extracellular matrix accumulation, tissue contraction, scar formation and …