Abstract：
:As gene therapy begins to produce its first clinical successes, interest in ocular gene transfer has grown owing to the favorable safety and efficacy characteristics of the eye as a target organ for drug delivery. Important advances also include the availability of viral and non-viral vectors that are able to efficiently transduce various ocular cell types, the use of intraocular delivery routes and the development of transcriptional regulatory elements that allow sustained levels of gene transfer in small and large animal models after a single administration. Here, we review recent progress in the field of ocular gene therapy. The first experiments in humans with severe inherited forms of blindness seem to confirm the good safety and efficacy profiles observed in animal models and suggest that gene transfer has the potential to become a valuable therapeutic strategy for otherwise untreatable blinding diseases.
journal_name
Trends Mol Medjournal_title
Trends in molecular medicineauthors
Colella P,Cotugno G,Auricchio Adoi
10.1016/j.molmed.2008.11.003subject
Has Abstractpub_date
2009-01-01 00:00:00pages
23-31issue
1eissn
1471-4914issn
1471-499Xpii
S1471-4914(08)00216-5journal_volume
15pub_type
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