부산대학교 도서관

Inherited retinal dystrophies (IRDs) constitute a heterogeneous group of retinal diseases that cause the progressive loss of vision. They are mainly associated with photoreceptor (rods and cones) or retinal pigment epithelium (RPE) dysfunction and loss. Several therapeutic strategies are ongoing in animal models and in patients, including gene, cell, or pharmacological therapies. However, at present, there is only one effective treatment available, Luxturna. The eye presents some characteristics that facilitate the development of different therapeutic strategies. It is an immune privilege and easily accessible organ. In the case of retinal diseases, one of the biggest challenges is the effective drug delivery to the diseased area because of the existence of several physiological and anatomical barriers of the eye. Among others, the routes for drug delivery to the retina include: 1) Topical that is a non‐invasive route, but does not provide the delivery of significant levels of therapeutic agents; 2) Systemic that can deliver a high level of therapeutic agents, but the blood‐retina barrier limits those that can be delivered; and 3) Intravitreal that provides a good delivery but can induce retinal endophthalmitis, retinal detachment, increased intraocular pressure or retinal haemorrhages. Nanotechnology has reformed the field of drug delivery and revolutionized medicine. Drug Delivery Systems (DDS) protect drugs from degradation, improve their permeability across cell membranes, their bioavailability, provide stable formulation and yield sustained and controlled release of drugs. Many types of nanoparticles (range in size from 10 to 1000 nm) used as drug carriers are made of polymers, lipids, inorganic metallic components, etc. DDS can overcome the limitations associated with current ocular therapy and ensure targeted and controlled drug delivery. We will present different nanocarriers loaded with therapeutic agents for the treatment of inherited retinal dystrophies. [ABSTRACT FROM AUTHOR]