Cystic fibrosis: Present and future

Digestion

Volumn 59, Issue 5, 1998, Pages 579-588

  Milla, P J ^a  

^a GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

Colonopathy; Cystic fibrosis; Fibrosing; Gene therapy; Genotype; Nutrition; Transplantation

Indexed keywords

PANCREAS ENZYME; TRANSMEMBRANE CONDUCTANCE REGULATOR;

CHLORIDE TRANSPORT; CHROMOSOME 7; COLON DISEASE; CYSTIC FIBROSIS; DISEASE CARRIER; DRUG MEGADOSE; ENZYME THERAPY; EPITHELIUM; EXOCRINE GLAND; GENE MUTATION; GENE THERAPY; GENOTYPE; GROWTH; HUMAN; INTESTINE; LIVER; LIVER FAILURE; LIVER TRANSPLANTATION; LUNG; LUNG INSUFFICIENCY; LUNG TRANSPLANTATION; MALNUTRITION; NUTRITION; PANCREAS; PANCREAS INSUFFICIENCY; PRENATAL DIAGNOSIS; PRIORITY JOURNAL; REVIEW; SURVIVAL;

CHROMOSOME ABERRATIONS; CHROMOSOME DISORDERS; CHROMOSOMES, HUMAN, PAIR 7; CYSTIC FIBROSIS; DIAGNOSIS, DIFFERENTIAL; GENE THERAPY; GENOTYPE; HUMANS; LIVER TRANSPLANTATION; LUNG TRANSPLANTATION; NUTRITIONAL SUPPORT;

EID: 0031926976     PISSN: 00122823     EISSN: None     Source Type: Journal    
DOI: 10.1159/000007533     Document Type: Review

References (46)

1. Welsh MJ, Tsui L-C, Boat TF, et al: Cystic fibrosis; in Scriver CR, Beaudet AL, Sly WS, et al (eds): Metabolic Basis of Inherited Disease, ed 7. New York, McGraw-Hill, 1995, pp 3799-3819.
2. MacLusky I, McLaughlin F, Levison H: Cystic fibrosis. Curr Prob Pediatr 1985;15:1-49.
3. Denning CR, Huang NN, Cusay LR, et al: Co-operative study comparing three methods of performing sweat tests to diagnose cystic fibrosis. Pediatrics 1980;66:752.
4. Riordan JR, Rommens JM, Kerem B, et al: Identification of the cystic fibrosis gene: Cloning and characterization of complementary DA. Science 1989;245:1066-1073.
5. Marino CR, Gorelick FS: Scientific advances in cystic fibrosis. Gastroenterology 1992;103: 681-683.
6. Lucotte G, Loirat F: A more detailed map of the cystic fibrosis mutation delta F508 frequencies in Europe. Hum Biol 1993;65:503-507.
7. Tsui L-C: The spectrum of cystic fibrosis mutations. Trend Genet 1992;8:392-396.
8. Welsh MJ, Smith AE: Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 1993;73:1251-1255.
9. Marino CR, Matovcik LM, Gorelick FS: Localization of the cystic fibrosis transmembrane conductance regulator in pancreas. J Clin Invest 1991;88:712-715.
10. Waters DL, Dorney SFA, Gaskin KJ, et al: Pancreatic function in infants identified as having cystic fibrosis in a neonatal screening program. N Engl J Med 1990;322:303-305.
11. Kristidis P, Bozon D, Corey M, et al: Genetic determination of exocrine pancreatic function in cystic fibrosis. Am J Hum Genet 1992;50: 1178-1183.
12. Johnson LG, Olsen JC, Sarkadi B, et al: Efficiency of great transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992;2:21-25.
13. Flotte TR: Prospects for virus-based gene therapy for cystic fibrosis. J Bioenerg Biomembr 1993;25:37-45.
14. Johnson LG: Gene therapy for cystic fibrosis. Chest 1995;107:77S.
15. O'Neal WK, Beaudet AL: Somatic gene therapy for cystic fibrosis. Hum Mol Genet 1994;3: 1497-1499.
16. Yang Y, Raper SE, Cohn JA, et al: An approach for treating the hepatobiliary disease of cystic fibrosis by somatic gene transfer. Proc Natl Acad Sci USA 1993;90:4601-4604.
17. Maeda H, Danel C, Crystal RG: Adenovirus-mediated transfer of human lipase complementary DNA to the gallbladder. Gastroenterology 1994;106:1638-1643.
18. Crystal RG, McElvaney NG, Rosenfeld MA, et al: Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract at individuals with cystic fibrosis. Nat Genet 1994;8:42-45.
19. Knowles MR, Hohneker KW, Zhou Zhaoging, et al: A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 1995;333:823-826.
20. Alton EWFW, Middleton PG, Caplen NJ, et al: Non-invasive liposome-mediated gene delivery can correct the ion transport defect in the cystic fibrosis mutant mice. Nat Genet 1993;5:135.
21. Gaskin KJ, Durie PR, Lee L, et al: Colipase and lipase secretion in childhood-onset pancreatic insufficiency: Delineation of patients with steatorrhoea secondary to relative colipase deficiency. Gastroenterology 1984;86:1-7.
22. Park RW, Grand RJ: Gastrointestinal manifestations of cystic fibrosis: A review. Gastroenterology 1981;81:1143-1156.
23. FitzSimmons SC: The changing epidemiology of cystic fibrosis. J Pediatr 1993;122:1-12.
24. Krueger LJ, Lerner A, Katz SM, et al: Cystic fibrosis and diabetes mellitus: Interactive or idiopathic. J Pediatr Gastroenterol Nutr 1991; 13:209-212.
25. Moran A, Diem P, Klein D, et al: Pancreatic endocrine function in cystic fibrosis. J Pediatr 1991;118:715-718.
26. Scott RB, O'Loughlin EV, Gall DG: Gastroesophageal reflux in patients with cystic fibrosis. J Pediatr 1985;106:223-226.
27. Gaskin KJ, Gurwitz D, Durie PR, et al: Improved respiratory prognosis in patients with cystic fibrosis with normal fat absorption. J Pediatr 1982;100:857-863.
28. Donnison AB, Shwachman H, Gross RE: A review of 164 children with meconium ileus seen at the Children's Hospital Medical Center, Boston. Pediatrics 1966;37:833-841.
29. Quinlan M, Way N, Joske R: Adult cystic fibrosis presenting with meconium ileus equivalent and diagnosed by selenomethionine scanning of the pancreas. Aust Ann Med 1967;16:84-86.
30. Cleghorn GJ, Forstner GG, Stringer DA, et al: Treatment of distal intestinal obstruction syndrome in cystic fibrosis with a balanced intestinal lavage solution. Lancet 1986;i:8.
31. Smith RL, van Velzen D, Smyth AR, Lloyd DA, Heaf DP: Strictures of ascending colon in cystic fibrosis and high-strength pancreatic enzymes. Lancet 1994;343:85-86.
32. Knabe N, Zak M, Hansen A, et al: Extensive pathological changes of the colon in cystic fibrosis and high-strength pancreatic enzymes. Lancet 1994;343:1230.
33. Smyth RL, Ashby D, O'Hea U, et al: Fibrosing colonopathy in cystic fibrosis: Results of a case-control study. Lancet 1995;346:1247-1251.
34. Fitzsimmons SC, Burkhart GA, Borowitz D, Grand RJ, Hammerstrom T, Durie PR, Lloyd-Still JD, Lowenfels AB: High-dose pancreatic-enzyme supplements and fibrosing colonopathy in children with cystic fibrosis. Massachusetts Medical Society 1997;336:1283-1289.
35. Borowitz DS, Grand RJ, Durie PR: Use of pancreatic enzyme supplements for patients with cystic fibrosis in the context of fibrosing colonopathy. J Pediatr 1995;127:681-684.
36. Roberts WC: The hepatic cirrhosis of cystic fibrosis of the pancreas. Am J Med 1962;32: 324-327.
37. Vaisman N, Pencharz PB, Corey M, et al: Energy expenditure of patients with cystic fibrosis. J Pediatr 1987;111:496-499.
38. Gaskin KJ: The liver and biliary tract in cystic fibrosis; in Suchy FJ (ed): Liver Disease in Children. St Louis, Mosby, 1994, pp 705-712.
39. Hubbard VS, Dunn GD, di Santi' Agnese PA: Abnormal fatty acid composition of plasma lipids in cystic fibrosis. Lancet 1977;ii:1302-1305.
40. Ramsey BW, Farrell PM, Pencharz P, and the Consensus Committee: Nutritional assessment and management in cystic fibrosis. Consensus Conferences, vol 1, sect V, April 1990, p 1.
41. Shepherd RW, Holt TL, Thomas BJ, et al: Nutritional rehabilitation in cystic fibrosis. Controlled studies of effects on nutritional growth retardation, body protein turnover, and course of pulmonary disease. J Pediatr 1986; 109:788-791.
42. Kraemer P, Rudegerg A, Hadorn B, et al: Relative underweight in cystic fibrosis and its prognostic value. Acta Pediatr Scad 1978;67:33-39.
43. Levy L, Durie P, Pencharz PB, et al: Effects of long-term nutritional rehabilitation on body composition and clinical status in malnourished children and adolescents with cystic fibrosis. J Pediatr 1985;107:225-229.
44. de Leval RM, Smyth R, Whitehead B, et al: Heart and lung transplantation for terminal cystic fibrosis: a 41/2-year experience. J Thorac Cardiovasc Surg 1991;101:633-642.
45. Balfour-Lynn IM, Martin I, Whitehead BF, Rees PG, Elliott MJ, de Leval MR: Heart-lung transplantation for patients under 10 with cystic fibrosis. Arch Dis Childh 1997;76:38-40.
46. Couetil JP, Houssin DP, Soubrane O, et al: Combined lung and liver transplantation in patients with cystic fibrosis: A 41/2-year experience. J Thorac Cardiovasc Surg 1995;110: 1415-1423.