Ocular delivery of nucleic acids: antisense oligonucleotides, aptamers and siRNA.

Abstract:

:Nucleic acids have gained a lot of interest for the treatment of ocular diseases. The first to enter in clinic has been Vitravene an antisense oligonucleotide for the treatment of cytomegalovirus (CMV) infection and more recently, research on aptamers have led to the marketing of anti-vascular endothelial growth factor (VEGF) inhibitor (Macugen) for the treatment of age-related macular degeneration (AMD). The siRNAs appear very promising as they are very potent inhibitors of protein expression. Despite their potential, nucleic acids therapeutic targets of nucleic acid-based drugs are mainly located in the posterior segment of the eye requiring invasive administration which can be harmful if repeated. Their intracellular penetration in some cases needs to be enhanced. This is the reason why adequate delivery systems were designed either to insure cellular penetration, protection against degradation or to allow long-term delivery. A combination of both effects was also developed for an implantable system. In conclusion, the intraocular administration of nucleic acids offers interesting perspectives for the treatment of ocular diseases.

journal_name

Adv Drug Deliv Rev

authors

Fattal E,Bochot A

doi

10.1016/j.addr.2006.07.020

subject

Has Abstract

pub_date

2006-11-15 00:00:00

pages

1203-23

issue

11

eissn

0169-409X

issn

1872-8294

pii

S0169-409X(06)00161-X

journal_volume

58

pub_type

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