Ocular drug delivery has constantly challenged ophthalmologists and drug delivery
scientists due to various anatomical and physiological barriers. Static and dynamic ocular …

Leber congenital amaurosis (LCA) is a rare hereditary retinal degeneration caused by
mutations in more than a dozen genes. RPE65, one of these mutated genes, is highly …

Retinal gene therapy with adeno‐associated viral (AAV) vectors is safe and effective in
humans. However, AAV's limited cargo capacity prevents its application to therapies of …

Patient-derived induced pluripotent stem cells (iPSCs) hold great promise for autologous
cell replacement. However, for many inherited diseases, treatment will likely require genetic …

A new family of gene delivery vectors is synthesized consisting of a medium-size generation
PAMAM dendrimer (generation 5, with amine termini) core randomly linked at the periphery …

Objectives A vast number of blinding diseases have genetic aetiologies and may be treated
by molecular based therapies such as antisense oligonucleotides or short interfering RNA …

Age related macular degeneration (AMD) is the most common cause of blindness amongst
the elderly. Approximately 10% of AMD patients suffer from an advanced form of AMD …

Gene therapy to treat ocular disorders is still starting, and current therapies are primarily
experimental, with most human clinical trials still in research state, although beginning to …

Inherited retinal dystrophies (IRDs) are a clinically and genetically heterogeneous group of
diseases with more than 250 causative genes. The most common form is retinitis …

Sensorineural hearing loss (SNHL) affects millions of people. Genetic mutations play a large
and direct role in both congenital and late-onset cases of SNHL (eg, age-dependent hearing …