Non-viral retinal gene therapy: a review.
In the developed world, diseases of the retina are common causes of untreatable blindness. In many cases, a genetic component to the aetiology has been identified, making the development of gene-based treatments a logical long-term goal. The clinical strategy for retinal gene therapy broadly encompasses two distinct advantages over systemic drug delivery. First is that gene delivery can limit expression of a therapeutic protein to a specific target cell, which is rarely possible even with loc...Expand abstract
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