Irradiation damage to salivary glands is a common iatrogenic consequence of treatment for head and neck cancers. The subsequent lack of saliva production leads to many functional and quality-of-life problems for affected patients and there is no effective conventional therapy. To address this problem, we developed an in vivo gene therapy strategy involving viral vector-mediated transfer of the aquaporin-1 cDNA to irradiation-damaged glands and successfully tested it in two pre-clinical models (irradiated rats and miniature pigs), as well as demonstrated its safety in a large toxicology and biodistribution study. Thereafter, a clinical research protocol was developed that has received approval from all required authorities in the United States. Patients are currently being enrolled in this study.
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Baum, B.J. et al. (2009). Aquaporin-1 Gene Transfer to Correct Radiation-Induced Salivary Hypofunction. In: Beitz, E. (eds) Aquaporins. Handbook of Experimental Pharmacology, vol 190. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-540-79885-9_20
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DOI: https://doi.org/10.1007/978-3-540-79885-9_20
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