Corneal gene therapy.
| Title: | Corneal gene therapy. |
|---|---|
| Authors: | Klausner EA; Midwestern University Chicago College of Pharmacy, 555 31st Street, Downers Grove, IL 60515, United States. eklaus@midwestern.edu; Peer D; Chapman RL; Multack RF; Andurkar SV |
| Source: | Journal of controlled release : official journal of the Controlled Release Society [J Control Release] 2007 Dec 20; Vol. 124 (3), pp. 107-33. Date of Electronic Publication: 2007 Jul 04. |
| Publication Type: | Journal Article; Review |
| Language: | English |
| Journal Info: | Publisher: Elsevier Science Publishers Country of Publication: Netherlands NLM ID: 8607908 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-4995 (Electronic) Linking ISSN: 01683659 NLM ISO Abbreviation: J Control Release Subsets: MEDLINE |
| Imprint Name(s): | Original Publication: Amsterdam : Elsevier Science Publishers, 1984- |
| MeSH Terms: | Genetic Therapy*; Corneal Diseases/*therapy; Cornea/physiology ; Corneal Dystrophies, Hereditary/therapy ; Graft Rejection/prevention & control ; Animals ; Corneal Transplantation ; Gene Transfer Techniques ; Genetic Vectors ; Humans ; Models, Biological |
| Abstract: | Gene therapy to the cornea can potentially correct inherited and acquired diseases of the cornea. Factors that facilitate corneal gene delivery are the accessibility and transparency of the cornea, its stability ex vivo and the immune privilege of the eye. Initial corneal gene delivery studies characterized the relationship between intraocular modes of administration and location of reporter gene expression. The challenge of achieving effective topical gene transfer, presumably due to tear flow, blinking and low penetration of the vector through epithlelial tight junctions left no alternative but invasive administration to the anterior chamber and corneal stroma. DNA vaccination, RNA interference and gene transfer of cytokines, growth factors and enzymes modulated the corneal microenvironment. Positive results were obtained in preclinical studies for prevention and treatment of corneal graft rejection, neovascularization, haze and herpetic stromal keratitis. These studies, corneal gene delivery systems and modes of administration, and considerations regarding the choice of animal species used are the focus of this review. Opportunities in the field of corneal gene therapy lie in expanding the array of corneal diseases investigated and in the implementation of recent designs of safer vectors with reduced immunogenicity and longer duration of gene expression. |
| Number of References: | 225 |
| Entry Date(s): | Date Created: 20070821 Date Completed: 20071212 Latest Revision: 20121115 |
| Update Code: | 20260130 |
| DOI: | 10.1016/j.jconrel.2007.05.041 |
| PMID: | 17707107 |
| Database: | MEDLINE |
Journal Article; Review