[Viral and nonviral gene therapy for treatment of retinal diseases].
The development of gene therapeutic approaches offers new perspectives for the treatment of retinal diseases. The use of both, nonviral methods employing oligonucleotides as well as viral expression vectors provide the possibility to treat neovascularization defects and retinal degeneration, respectively. The mechanism by which the therapeutic oligonucleotides (antisense oligonucleotides, aptamers and siRNA) work is based on degradation of specific transcripts. Consequently, a reduction of the corresponding protein, which is involved in the particular pathogenesis, follows. In contrast, viral vector transduction can substitute the disease-associated gene with an intact copy. So far, animal models have successfully contributed to the development of gene therapeutic medication and further treatments are at the recruiting phase of clinical trials.
Neidhardt
J
J
Abteilung für Medizinische Molekulargenetik und Gendiagnostik des Instituts für Medizinische Genetik, Universität Zürich, Schwerzenbach, Schweiz.
Wycisk
K
K
Klöckener-Gruissem
B
B
D000818:Animals; D005136:Eye Proteins / Q000235:genetics*; D018390:Gene Targeting / Q000379:methods; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D009841:Oligonucleotides / Q000008:administration & dosage* / Q000235:genetics; D012164:Retinal Diseases / Q000235:genetics* / Q000628:therapy* / Q000821:virology; D014162:Transfection / Q000379:methods; D014780:Viruses / Q000235:genetics
D005136:Eye Proteins; D009841:Oligonucleotides