Aquaporin-1 gene transfer to correct radiation-induced salivary hypofunction.
Aquaporin-1 gene transfer to correct radiation-induced salivary hypofunction.
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DOI：
10.1007/978-3-540-79885-9_20
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发表时间：
2009
影响因子：
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通讯作者：
Illei, Gabor G
中科院分区：
文献类型：
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作者：
Baum, Bruce J;Zheng, Changyu;Cotrim, Ana P;McCullagh, Linda;Goldsmith, Corinne M;Brahim, Jaime S;Atkinson, Jane C;Turner, R James;Liu, Shuying;Nikolov, Nikolay;Illei, Gabor G
放射线对唾液腺的损伤是头颈部癌症治疗中常见的医源性后果。随后缺乏唾液产生导致受影响患者的许多功能和生活质量问题，并且没有有效的常规治疗。为了解决这个问题，我们开发了一种体内基因治疗策略，包括病毒载体介导的水通道蛋白-1 cDNA转移到辐射损伤的腺体，并成功地在两个临床前模型（辐射大鼠和小型猪）中进行了测试，并在大型毒理学和生物分布研究中证明了其安全性。此后，制定了一项临床研究方案，该方案已获得美国所有必要当局的批准。患者目前正在入组本研究。
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.