Ocular drug delivery: Molecules, cells, and genes

Department of Ophthalmology and Visual Sciences, University of Wisconsin, Madison 53705-3611, USA.
Canadian Journal of Ophthalmology (Impact Factor: 1.3). 07/2007; 42(3):447-54. DOI: 10.3129/i07-049
Source: PubMed

ABSTRACT Recent advances in molecular cell biology have led to the exploration of new therapies based on molecules, cells, and genes to treat a variety of ocular disorders. In this review, we present the current state of knowledge pertaining to the development of different delivery systems to mediate safe and long-lived therapies, with an emphasis on gene therapy. The advantages and limitations of these delivery and therapeutic methods are also discussed.

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    ABSTRACT: Glaucoma is a complex, life-long disease that requires an individualized, multifaceted approach to treatment. Most patients will be started on topical ocular hypotensive eyedrop therapy, and over time multiple classes of drugs will be needed to control their intraocular pressure. The search for drugs with novel mechanisms of action, to treat those who do not achieve adequate intraocular pressure control with, or become refractory to, current therapeutics, is ongoing, as is the search for more efficient, targeted drug delivery methods. Gene-transfer and stem-cell applications for glaucoma therapeutics are moving forward. Advances in imaging technologies improve our understanding of glaucoma pathophysiology and enable more refined patient evaluation and monitoring, improving patient outcomes.
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    ABSTRACT: RNA interference (RNAi) is a relatively novel technology that is currently being exploited for therapeutic purposes for several indications. As of today, there is no approved therapy based on this technology but several compounds are in advanced phases of clinical development. The eye was one of the first settings in which proof of concept was achieved; but in the latest years successful development of delivery technologies to the liver have made this organ the preferred target of therapies based on RNAi. We have used the results obtained in preclinical and clinical development of several compounds for different ocular conditions to demonstrate that the eye represents an ideal organ for therapies based on RNAi.
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