Whitcomb D C
Department of Medicine, University of Pittsburgh, Pennsylvania, USA.
Gastroenterol Clin North Am. 1999 Sep;28(3):525-41. doi: 10.1016/s0889-8553(05)70071-6.
Hereditary pancreatitis is an unusual form of acute and chronic pancreatitis that is usually associated with two specific mutations in the cationic trypsinogen gene. The extensive information available on the biochemistry, cell biology, and molecular biology of cationic trypsinogen and its gene provides the groundwork for development of a variety of therapeutic strategies, including gene therapy. Several features of this disease, however, make gene therapy unlikely in the near future. Further research using new models, including transgenic animals, is required before breakthroughs in therapy can be expected.
遗传性胰腺炎是一种不常见的急慢性胰腺炎形式，通常与阳离子胰蛋白酶原基因的两种特定突变相关。关于阳离子胰蛋白酶原及其基因的生物化学、细胞生物学和分子生物学的大量信息为包括基因治疗在内的多种治疗策略的开发奠定了基础。然而，这种疾病的几个特征使得基因治疗在近期不太可能实现。在有望取得治疗突破之前，需要使用包括转基因动物在内的新模型进行进一步研究。
