-
1
-
-
0024424270
-
Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA
-
Riordan JR, Rommens JM, Kerem B, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 1989; 245:1066-1073.
-
(1989)
Science
, vol.245
, pp. 1066-1073
-
-
Riordan, J.R.1
Rommens, J.M.2
Kerem, B.3
-
2
-
-
33745755929
-
Gene therapy progress and prospects: Cystic fibrosis
-
Summarizes current gene strategies
-
Griesebach U, Geddes D, Alton E. Gene therapy progress and prospects: cystic fibrosis. Gene Ther 2006; 13:1061-1067. Summarizes current gene strategies.
-
(2006)
Gene Ther
, vol.13
, pp. 1061-1067
-
-
Griesebach, U.1
Geddes, D.2
Alton, E.3
-
3
-
-
33846821729
-
Calculating expected lung deposition of aerosolized administration of AAV vector in human clinical studies
-
Leung K, Louca E, Munson K, et al. Calculating expected lung deposition of aerosolized administration of AAV vector in human clinical studies. J Gene Med 2007; 9:10-21.
-
(2007)
J Gene Med
, vol.9
, pp. 10-21
-
-
Leung, K.1
Louca, E.2
Munson, K.3
-
4
-
-
10744227749
-
Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial
-
Moss R, Rodman D, Spencer L, et al. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest 2004; 125:509-521.
-
(2004)
Chest
, vol.125
, pp. 509-521
-
-
Moss, R.1
Rodman, D.2
Spencer, L.3
-
5
-
-
33745222075
-
Mutation specific therapy in CF
-
Kerem E. Mutation specific therapy in CF. Paediatr Respir Rev 2006; 7:S166-S169.
-
(2006)
Paediatr Respir Rev
, vol.7
-
-
Kerem, E.1
-
6
-
-
0001752544
-
Cystic fibrosis
-
Scriver CR, Beaudet AL, Sly WS, Valle D, editors, New York: McGraw-Hill;
-
Welsh M, Ramsey BW, Accurso F, Cutting GR. Cystic fibrosis. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The molecular and metabolic basis of inherited disease. New York: McGraw-Hill; 2001. pp. 5121-5188.
-
(2001)
The molecular and metabolic basis of inherited disease
, pp. 5121-5188
-
-
Welsh, M.1
Ramsey, B.W.2
Accurso, F.3
Cutting, G.R.4
-
7
-
-
0034961464
-
Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis
-
Clancy J, Bebok Z, Ruiz F, et al. Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis. Am J Respir Crit Care Med 2001; 163:1683-1692.
-
(2001)
Am J Respir Crit Care Med
, vol.163
, pp. 1683-1692
-
-
Clancy, J.1
Bebok, Z.2
Ruiz, F.3
-
8
-
-
0141863491
-
Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations
-
Wilschanski M, Yahav Y, Yaacov Y, et al. Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations. N Engl J Med 2003; 349:1433-1441.
-
(2003)
N Engl J Med
, vol.349
, pp. 1433-1441
-
-
Wilschanski, M.1
Yahav, Y.2
Yaacov, Y.3
-
9
-
-
34247588271
-
PTC124 targets genetic disorders caused by nonsense mutations
-
Welch E, Barton E, Zhuo J, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007; 441:87-91.
-
(2007)
Nature
, vol.441
, pp. 87-91
-
-
Welch, E.1
Barton, E.2
Zhuo, J.3
-
10
-
-
34347224756
-
No detectable improvements in CFTR by nasal aminoglycosides in CF patients with stop mutations
-
Clancy J, Rowe S, Bebok Z, et al. No detectable improvements in CFTR by nasal aminoglycosides in CF patients with stop mutations. Am J Respir Cell Mol Biol 2007; 37:57-66.
-
(2007)
Am J Respir Cell Mol Biol
, vol.37
, pp. 57-66
-
-
Clancy, J.1
Rowe, S.2
Bebok, Z.3
-
11
-
-
33847360602
-
Nonsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin
-
Gives a functional explanation for the variability in response seen in patients following readthrough therapy
-
Linde L, Boelz S, Nissim-Rafinia M, et al. Nonsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin. J Clin Invest 2007; 117:683-692. Gives a functional explanation for the variability in response seen in patients following readthrough therapy.
-
(2007)
J Clin Invest
, vol.117
, pp. 683-692
-
-
Linde, L.1
Boelz, S.2
Nissim-Rafinia, M.3
-
12
-
-
34247200483
-
In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: A pilot study
-
Sermet-Gaudelus I, Renouil M, Fajac A, et al. In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study. BMC Med 2007; 5:5.
-
(2007)
BMC Med
, vol.5
, pp. 5
-
-
Sermet-Gaudelus, I.1
Renouil, M.2
Fajac, A.3
-
13
-
-
0025242929
-
Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis
-
Cheng S, Gregory R, Marshall J, et al. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis. Cell 1990; 63:827-834.
-
(1990)
Cell
, vol.63
, pp. 827-834
-
-
Cheng, S.1
Gregory, R.2
Marshall, J.3
-
14
-
-
33745190973
-
Selective inhibition of endoplasmic reticulum-associated degradation rescues DeltaF508-cystic fibrosis transmembrane regulator and suppresses interleukin-8 levels: Therapeutic implications
-
Vij N, Fang S, Zeitlin P. Selective inhibition of endoplasmic reticulum-associated degradation rescues DeltaF508-cystic fibrosis transmembrane regulator and suppresses interleukin-8 levels: therapeutic implications. J Biol Chem 2006; 281:17369-17378.
-
(2006)
J Biol Chem
, vol.281
, pp. 17369-17378
-
-
Vij, N.1
Fang, S.2
Zeitlin, P.3
-
15
-
-
0036665609
-
Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate
-
Zeitlin P, Diener-West M, Rubenstein R, et al. Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate. Mol Ther 2002; 6:119-126.
-
(2002)
Mol Ther
, vol.6
, pp. 119-126
-
-
Zeitlin, P.1
Diener-West, M.2
Rubenstein, R.3
-
16
-
-
11144355340
-
Curcumin, a major constituent of turmeric, corrects cystic fibrosis defects
-
Egan M, Pearson M, Weiner SA, et al. Curcumin, a major constituent of turmeric, corrects cystic fibrosis defects. Science 2004; 304:600-602.
-
(2004)
Science
, vol.304
, pp. 600-602
-
-
Egan, M.1
Pearson, M.2
Weiner, S.A.3
-
17
-
-
6044268094
-
Curcumin does not stimulate cAMP-mediated chloride transport in cystic fibrosis airway epithelial cells
-
Dragomir A, Bjorstad J, Hjelte L, Roomans G. Curcumin does not stimulate cAMP-mediated chloride transport in cystic fibrosis airway epithelial cells. Biochem Biophys Res Commun 2006; 322:447-451.
-
(2006)
Biochem Biophys Res Commun
, vol.322
, pp. 447-451
-
-
Dragomir, A.1
Bjorstad, J.2
Hjelte, L.3
Roomans, G.4
-
18
-
-
4644360693
-
Evidence against the rescue of defective DeltaF508-CFTR cellular processing by curcumin in cell culture and mouse models
-
Song Y, Sonawane N, Salinas D, et al. Evidence against the rescue of defective DeltaF508-CFTR cellular processing by curcumin in cell culture and mouse models. J Biol Chem 2004; 279:40629-40633.
-
(2004)
J Biol Chem
, vol.279
, pp. 40629-40633
-
-
Song, Y.1
Sonawane, N.2
Salinas, D.3
-
19
-
-
33947543364
-
Curcumin opens cystic fibrosis transmembrane conductance regulator channels by a novel mechanism that requires neither ATP binding nor dimerization of the nucleotide-binding domains
-
Wang W, Bernard K, Li G, Kirk K. Curcumin opens cystic fibrosis transmembrane conductance regulator channels by a novel mechanism that requires neither ATP binding nor dimerization of the nucleotide-binding domains. J Biol Chem 2006; 282:4533-4544.
-
(2006)
J Biol Chem
, vol.282
, pp. 4533-4544
-
-
Wang, W.1
Bernard, K.2
Li, G.3
Kirk, K.4
-
20
-
-
24644464284
-
Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening
-
Pedemonte N, Lukacs G, Du K, et al. Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening. J Clin Invest 2001; 115:2564-2571.
-
(2001)
J Clin Invest
, vol.115
, pp. 2564-2571
-
-
Pedemonte, N.1
Lukacs, G.2
Du, K.3
-
21
-
-
33744831154
-
Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
-
Van Goor F, Straley K, Cao D, et al. Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Cell Physiol 2006; 290:L1117-L1130.
-
(2006)
Am J Physiol Cell Physiol
, vol.290
-
-
Van Goor, F.1
Straley, K.2
Cao, D.3
-
22
-
-
27644594653
-
Proteasome inhibition induces selective motor neuron death in organotypic slice cultures
-
Tsuji S, Kikuchi S, Shinpo K, et al. Proteasome inhibition induces selective motor neuron death in organotypic slice cultures. J Neurosci Res 2005; 82:443-451.
-
(2005)
J Neurosci Res
, vol.82
, pp. 443-451
-
-
Tsuji, S.1
Kikuchi, S.2
Shinpo, K.3
-
24
-
-
27444444705
-
Normal and cystic fibrosis airway surface liquid homeostasis: The effects of phasic shear stress and viral infections
-
Tarran R, Button B, Picher M, et al. Normal and cystic fibrosis airway surface liquid homeostasis: the effects of phasic shear stress and viral infections. J Biol Chem 2005; 280:35751-35759.
-
(2005)
J Biol Chem
, vol.280
, pp. 35751-35759
-
-
Tarran, R.1
Button, B.2
Picher, M.3
-
25
-
-
0025874599
-
Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis
-
Knowles M, Clarke L, Boucher R. Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis. N Engl J Med 1991; 3252:533-538.
-
(1991)
N Engl J Med
, vol.3252
, pp. 533-538
-
-
Knowles, M.1
Clarke, L.2
Boucher, R.3
-
26
-
-
0035880177
-
Effect of aerosolized uridine 5′-triphosphate on mucociliary clearance in mild chronic bronchitis
-
Bennett W, Zeman K, Foy C, et al. Effect of aerosolized uridine 5′-triphosphate on mucociliary clearance in mild chronic bronchitis. Am J Respir Crit Care Med 2001; 164:302-306.
-
(2001)
Am J Respir Crit Care Med
, vol.164
, pp. 302-306
-
-
Bennett, W.1
Zeman, K.2
Foy, C.3
-
27
-
-
20144363443
-
Cystic Fibrosis Foundation Therapeutics Development Network. Safety and tolerability of denufosol tetrasodium inhalation solution, a novel P2Y2 receptor agonist: Results of a phase 1/phase 2 multicenter study in mild to moderate cystic fibrosis
-
Deterding R, Retsch-Bogart G, Milgram L, et al. Cystic Fibrosis Foundation Therapeutics Development Network. Safety and tolerability of denufosol tetrasodium inhalation solution, a novel P2Y2 receptor agonist: results of a phase 1/phase 2 multicenter study in mild to moderate cystic fibrosis. Pediatr Pulmonol 2005; 39:339-348.
-
(2005)
Pediatr Pulmonol
, vol.39
, pp. 339-348
-
-
Deterding, R.1
Retsch-Bogart, G.2
Milgram, L.3
-
28
-
-
34547949974
-
Phase 2 randomized safety and efficacy trial of nebulized denufosol tetrasodium in cystic fibrosis
-
19 April [Epub ahead of print, Clinical study on a new approach to circumvent the CFTR defect
-
Deterding R, Lavange L, Engels J, et al. Phase 2 randomized safety and efficacy trial of nebulized denufosol tetrasodium in cystic fibrosis. Am J Respir Crit Care Med 2007; 19 April [Epub ahead of print]. Clinical study on a new approach to circumvent the CFTR defect.
-
(2007)
Am J Respir Crit Care Med
-
-
Deterding, R.1
Lavange, L.2
Engels, J.3
-
29
-
-
1642493889
-
A phase I trial of intranasal Moli1901 for cystic fibrosis
-
Zeitlin P, Boyle M, Guggino W. A phase I trial of intranasal Moli1901 for cystic fibrosis. Chest 2005; 25:143-149.
-
(2005)
Chest
, vol.25
, pp. 143-149
-
-
Zeitlin, P.1
Boyle, M.2
Guggino, W.3
-
30
-
-
34248597371
-
-
Grasemann H, Stehling F, Brunar H, et al. Inhalation of moli1901 in patients with cystic fibrosis. Chest 2007; 131:1461-1466. Clinical study showing the efficacy on lung function of a non-CFTR-mediated pharmacotherapy.
-
Grasemann H, Stehling F, Brunar H, et al. Inhalation of moli1901 in patients with cystic fibrosis. Chest 2007; 131:1461-1466. Clinical study showing the efficacy on lung function of a non-CFTR-mediated pharmacotherapy.
-
-
-
-
31
-
-
0033581885
-
Activation of the epithelial Na+ channel (ENaC) requires CFTR Cl- channel function
-
Reddy M, Light MJ, Quinton P. Activation of the epithelial Na+ channel (ENaC) requires CFTR Cl- channel function. Nature 1999; 402:301-304.
-
(1999)
Nature
, vol.402
, pp. 301-304
-
-
Reddy, M.1
Light, M.J.2
Quinton, P.3
-
32
-
-
2442718786
-
Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice
-
Mall M, Grubb B, Harkema J, et al. Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice. Nat Med 2004; 10:487-493.
-
(2004)
Nat Med
, vol.10
, pp. 487-493
-
-
Mall, M.1
Grubb, B.2
Harkema, J.3
-
34
-
-
30944452384
-
Mucus clearance and lung function in cystic fibrosis with hypertonic saline
-
Donaldson S, Bennett W, Zeman K, et al. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med 2006; 354:241-250.
-
(2006)
N Engl J Med
, vol.354
, pp. 241-250
-
-
Donaldson, S.1
Bennett, W.2
Zeman, K.3
-
35
-
-
33748744717
-
Hypertonic saline therapy in cystic fibrosis: Evidence against the proposed mechanism involving aquaporins
-
Levin M, Sullivan S, Nielson D, et al. Hypertonic saline therapy in cystic fibrosis: evidence against the proposed mechanism involving aquaporins. J Biol Chem 2006; 281:25803-25812.
-
(2006)
J Biol Chem
, vol.281
, pp. 25803-25812
-
-
Levin, M.1
Sullivan, S.2
Nielson, D.3
-
36
-
-
33745838632
-
Design, synthesis, and structure-activity relationships of novel 2-substituted pyrazinoylguanidine epithelial sodium channel blockers: Drugs for cystic fibrosis and chronic bronchitis
-
Hirsh A, Molino B, Zhang J, et al. Design, synthesis, and structure-activity relationships of novel 2-substituted pyrazinoylguanidine epithelial sodium channel blockers: drugs for cystic fibrosis and chronic bronchitis. J Med Chem 2006; 49:4098-4115.
-
(2006)
J Med Chem
, vol.49
, pp. 4098-4115
-
-
Hirsh, A.1
Molino, B.2
Zhang, J.3
-
37
-
-
30944452609
-
Restoring airway surface liquid in cystic fibrosis
-
Ratjen F. Restoring airway surface liquid in cystic fibrosis. N Engl J Med 2006; 354:291-293.
-
(2006)
N Engl J Med
, vol.354
, pp. 291-293
-
-
Ratjen, F.1
-
38
-
-
0030784262
-
Effect of increasing doses of hypertonic saline on mucociliary clearance in patients with cystic fibrosis
-
Robinson M, Hemming A, Regnis J, et al. Effect of increasing doses of hypertonic saline on mucociliary clearance in patients with cystic fibrosis. Thorax 1997; 52:900-903.
-
(1997)
Thorax
, vol.52
, pp. 900-903
-
-
Robinson, M.1
Hemming, A.2
Regnis, J.3
-
39
-
-
30944466084
-
National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis
-
Elkins M, Robinson M, Rose B, et al., National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med 2006; 354:229-240.
-
(2006)
N Engl J Med
, vol.354
, pp. 229-240
-
-
Elkins, M.1
Robinson, M.2
Rose, B.3
-
40
-
-
34247880282
-
Pilot study of safety and tolerability of inhaled hypertonic saline in infants with cystic fibrosis
-
Subbarao P, Balkovec S, Solomon M, Ratjen F. Pilot study of safety and tolerability of inhaled hypertonic saline in infants with cystic fibrosis. Pediatr Pulmonol 2007; 42:471-476.
-
(2007)
Pediatr Pulmonol
, vol.42
, pp. 471-476
-
-
Subbarao, P.1
Balkovec, S.2
Solomon, M.3
Ratjen, F.4
-
41
-
-
0032834277
-
The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: A pilot study
-
Robinson M, Daviskas E, Eberl S, et al. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J 1999; 14:678-685.
-
(1999)
Eur Respir J
, vol.14
, pp. 678-685
-
-
Robinson, M.1
Daviskas, E.2
Eberl, S.3
-
42
-
-
0033386691
-
Cystic fibrosis lung disease: The role of nitric oxide
-
Grasemann H, Ratjen F. Cystic fibrosis lung disease: the role of nitric oxide. Pediatr Pulmonol 1999; 28:442-448.
-
(1999)
Pediatr Pulmonol
, vol.28
, pp. 442-448
-
-
Grasemann, H.1
Ratjen, F.2
-
43
-
-
34250835575
-
Nitrosative stress inhibits production of the virulence factor alginate in mucoid Pseudomonas aeruginosa
-
Wood S, Firoved A, Ornatowski W, et al. Nitrosative stress inhibits production of the virulence factor alginate in mucoid Pseudomonas aeruginosa. Free Radic Res 2007; 41:208-215.
-
(2007)
Free Radic Res
, vol.41
, pp. 208-215
-
-
Wood, S.1
Firoved, A.2
Ornatowski, W.3
-
44
-
-
32444449521
-
Anaerobic killing of mucoid Pseudomonas aeruginosa by acidified nitrite derivatives under cystic fibrosis airway conditions
-
Yoon S, Coakley R, Lau G, et al. Anaerobic killing of mucoid Pseudomonas aeruginosa by acidified nitrite derivatives under cystic fibrosis airway conditions. J Clin Invest 2006; 116:436-446.
-
(2006)
J Clin Invest
, vol.116
, pp. 436-446
-
-
Yoon, S.1
Coakley, R.2
Lau, G.3
-
45
-
-
33745761197
-
Inhaled L-arginine improves exhaled nitric oxide and pulmonary function in patients with cystic fibrosis
-
Augmentation of nitric oxide production improved lung function and oxygen saturation in cystic fibrosis patients
-
Grasemann H, Kurtz F, Ratjen F. Inhaled L-arginine improves exhaled nitric oxide and pulmonary function in patients with cystic fibrosis. Am J Respir Crit Care Med 2006; 174:208-212. Augmentation of nitric oxide production improved lung function and oxygen saturation in cystic fibrosis patients.
-
(2006)
Am J Respir Crit Care Med
, vol.174
, pp. 208-212
-
-
Grasemann, H.1
Kurtz, F.2
Ratjen, F.3
-
46
-
-
33744461829
-
Anti-inflammatory effect of augmented nitric oxide production in chronic lung infection
-
Hopkins N, Gunning Y, O'Croinin D, et al. Anti-inflammatory effect of augmented nitric oxide production in chronic lung infection. J Pathol 2006; 209:198-205.
-
(2006)
J Pathol
, vol.209
, pp. 198-205
-
-
Hopkins, N.1
Gunning, Y.2
O'Croinin, D.3
-
47
-
-
33748944330
-
S-nitrosylating agents: A novel class of compounds that increase cystic fibrosis
-
Zaman K, Carraro S, Doherty J, et al. S-nitrosylating agents: a novel class of compounds that increase cystic fibrosis. Mol Pharmacol 2006; 70:1435-1442.
-
(2006)
Mol Pharmacol
, vol.70
, pp. 1435-1442
-
-
Zaman, K.1
Carraro, S.2
Doherty, J.3
-
49
-
-
33745686502
-
Microbiology, safety, and pharmacokinetics of aztreonam lysinate for inhalation in patients with cystic fibrosis
-
Gibson RL, Retsch-Bogart GZ, Oermann C, et al. Microbiology, safety, and pharmacokinetics of aztreonam lysinate for inhalation in patients with cystic fibrosis. Pediatr Pulmonol 2006; 41:656-665.
-
(2006)
Pediatr Pulmonol
, vol.41
, pp. 656-665
-
-
Gibson, R.L.1
Retsch-Bogart, G.Z.2
Oermann, C.3
|