Gene therapy, 2009-08, Vol.16 (8), p.982-989
2009
Details
- Autor(en) / Beteiligte
- Titel
- Therapeutic silencing of an endogenous gene by siRNA cream in an arthritis model mouse
- Ist Teil von
- Gene therapy, 2009-08, Vol.16 (8), p.982-989
- Ort / Verlag
- London: Nature Publishing Group
- Erscheinungsjahr
- 2009
- Link zum Volltext
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- Recent advances of biotechnology have laid the groundwork for potent and specific molecular-targeting therapies including RNA interference. The largest remaining hurdle for widespread use of this technology in skin is an effective delivery system. Here, we demonstrate an effective topical delivery system using a cream formulation containing a small-interfering RNA (siRNA) that specifically targets osteopontin (OPN). OPN is a validated target in numerous inflammatory diseases, including rheumatoid arthritis (RA). The siRNA targeting OPN was incorporated into a cream formulation GeneCream that penetrates the stratum corneum, depositing siRNA in the epidermis, dermis, and to a lesser extent, subcutaneous tissue. In addition, when the OPN siRNA cream was topically applied to the skin of a collagen antibody-induced RA mouse model, the siRNA cream prevented the occurrence of severe, irreversible damage to bone and cartilage. Thus, the siRNA cream provides effective delivery of active OPN siRNA, suggesting this formulation may represent a platform technology for delivery of siRNAs for treating various disorders including RA.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0969-7128eISSN: 1476-5462DOI: 10.1038/gt.2009.66Titel-ID: cdi_proquest_miscellaneous_733965428
- Format
- –
- Schlagworte
- Administration, Topical, Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy, Animals, Applied cell therapy and gene therapy, Arthritis, Arthritis, Experimental - therapy, Arthritis, Rheumatoid - therapy, Biological and medical sciences, Biotechnology, Care and treatment, Cartilage, Collagen, Cytokines, Dermis, Epidermis, Female, Fundamental and applied biological sciences. Psychology, Gene expression, Gene Expression Regulation, Gene Targeting - methods, Gene therapy, Gene Transfer Techniques, Genetic aspects, Genetic Therapy - methods, Genetic transcription, Health. Pharmaceutical industry, Industrial applications and implications. Economical aspects, Inflammatory diseases, Medical sciences, Methods, Mice, Mice, Inbred BALB C, Molecular biology, Ointments - adverse effects, Osteopontin, Osteopontin - genetics, Osteopontin - metabolism, Physiological aspects, Rheumatoid arthritis, RNA Interference, RNA, Small Interfering - administration & dosage, RNA-mediated interference, Rodents, siRNA, Skin, Skin Absorption, Stratum corneum, Transdermal medication, Transfusions. Complications. Transfusion reactions. Cell and gene therapy