NR 3-4/2002

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Perspektywy terapii genowej w okulistyce
Gene therapy prospects in ophthalmology
Marcin Stopa
Z Katedry i Kliniki Okulistyki Akademii Medycznej im. Karola Marcinkowskiego w Poznaniu
Kierownik: prof. dr hab. n. med. Krystyna Pecold |
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| Summary: |
Purpose: Presentation of newest
achievements from borderland of ophthalmology and molecular biology and their clinical
employment areas in the therapy of eye diseases.
Material and methods: MEDLINE database has been searched for terms gene,
gene therapy, ocular, eye in title and summary fields.
Results: Based on the latest literature eye disorders have been selected
that are currently in focus of gene therapy. Experimental approaches in vitro as well as
in vivo have been reviewed.
Conclusions: modern ophthalmology profits more and more from newest
achievements in molecular biology such as gene therapy. Transfer of additional genes to
selected target cells within eye is able to change function and allows to achieve required
effects. It is performed through employment of so called vectors, small particles allowing
the gene to be transferred. Presently most often used vectors are derived from viruses
(retrovirus, adenovirus, viruses AAV). Experimental studies have been performed in
following eye diseases: retinitis pigmentosa, Leber congenital amaurosis, glaucoma,
retinoblastoma, postoperative posterior capsule opacification, corneal diseases, age
related macular degeneration, uveal melanoma. In each case the experimental results were
promising. However, possible side effects of such therapy are not sufficiently known.
There have been no clinical gene therapy studies in ophthalmology so far. |
| Słowa kluczowe: |
transfer genów, terapia genowa, oko, wektor. |
| Key words: |
gene transfer, gene therapy, eye, vector. |
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