The spectrum of complications of hereditary pancreatitis. Is this a model for future gene therapy?
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.
Whitcomb
D C
DC
Department of Medicine, University of Pittsburgh, Pennsylvania, USA.
D000595:Amino Acid Sequence; D000818:Animals; D002919:Chymotrypsinogen / Q000235:genetics; D015316:Genetic Therapy; D006801:Humans; D008969:Molecular Sequence Data; D009154:Mutation; D010190:Pancreatic Neoplasms / Q000209:etiology / Q000235:genetics / Q000517:prevention & control; D010195:Pancreatitis / Q000150:complications* / Q000235:genetics* / Q000378:metabolism / Q000628:therapy; D014362:Trypsinogen / Q000235:genetics
D014362:Trypsinogen; D002919:Chymotrypsinogen