Novel Pharmacologic Therapies for Cystic Fibrosis

Overview

Journal J Clin Invest

Specialty General Medicine

Date 1999 Feb 18

PMID 10021451

Citations 9

Authors

P L Zeitlin

Affiliations

Soon will be listed here.

Citing Articles

Transcriptomic and Proteostasis Networks of CFTR and the Development of Small Molecule Modulators for the Treatment of Cystic Fibrosis Lung Disease.

Strub M, McCray Jr P Genes (Basel). 2020; 11(5).

PMID: 32414011 PMC: 7288469. DOI: 10.3390/genes11050546.

Interference with ubiquitination in CFTR modifies stability of core glycosylated and cell surface pools.

Lee S, Henderson M, Schiffhauer E, Despanie J, Henry K, Kang P Mol Cell Biol. 2014; 34(14):2554-65.

PMID: 24777605 PMC: 4097669. DOI: 10.1128/MCB.01042-13.

The iodide-transport-defect-causing mutation R124H: a δ-amino group at position 124 is critical for maturation and trafficking of the Na+/I- symporter.

Paroder V, Nicola J, Ginter C, Carrasco N J Cell Sci. 2013; 126(Pt 15):3305-13.

PMID: 23690546 PMC: 3730242. DOI: 10.1242/jcs.120246.

Applications of proteomic technologies for understanding the premature proteolysis of CFTR.

Henderson M, Singh O, Zeitlin P Expert Rev Proteomics. 2010; 7(4):473-86.

PMID: 20653504 PMC: 2924573. DOI: 10.1586/epr.10.42.

Diagnosis of cystic fibrosis.

Voter K, Ren C Clin Rev Allergy Immunol. 2008; 35(3):100-6.

PMID: 18506640 DOI: 10.1007/s12016-008-8078-x.

References

Jensen T, Loo M, Pind S, Williams D, GOLDBERG A, Riordan J . Multiple proteolytic systems, including the proteasome, contribute to CFTR processing. Cell. 1995; 83(1):129-35. DOI: 10.1016/0092-8674(95)90241-4. View

Illek B, Fischer H, Machen T . Genetic disorders of membrane transport. II. Regulation of CFTR by small molecules including HCO3-. Am J Physiol. 1998; 275(6):G1221-6. DOI: 10.1152/ajpgi.1998.275.6.G1221. View

Howard M, Frizzell R, Bedwell D . Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations. Nat Med. 1996; 2(4):467-9. DOI: 10.1038/nm0496-467. View

Smith J, Travis S, Greenberg E, Welsh M . Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell. 1996; 85(2):229-36. DOI: 10.1016/s0092-8674(00)81099-5. View

Ma J, Tasch J, Tao T, Zhao J, Xie J, Drumm M . Phosphorylation-dependent block of cystic fibrosis transmembrane conductance regulator chloride channel by exogenous R domain protein. J Biol Chem. 1996; 271(13):7351-6. DOI: 10.1074/jbc.271.13.7351. View

Ismailov I, Awayda M, Jovov B, Berdiev B, Fuller C, Dedman J . Regulation of epithelial sodium channels by the cystic fibrosis transmembrane conductance regulator. J Biol Chem. 1996; 271(9):4725-32. DOI: 10.1074/jbc.271.9.4725. View

Haws C, Nepomuceno I, Krouse M, Wakelee H, Law T, Xia Y . Delta F508-CFTR channels: kinetics, activation by forskolin, and potentiation by xanthines. Am J Physiol. 1996; 270(5 Pt 1):C1544-55. DOI: 10.1152/ajpcell.1996.270.5.C1544. View

Brown C, Welch W . Correcting temperature-sensitive protein folding defects. J Clin Invest. 1997; 99(6):1432-44. PMC: 507959. DOI: 10.1172/JCI119302. View

Cohen B, Lee G, Jacobson K, Kim Y, Huang Z, Sorscher E . 8-cyclopentyl-1,3-dipropylxanthine and other xanthines differentially bind to the wild-type and delta F508 first nucleotide binding fold (NBF-1) domains of the cystic fibrosis transmembrane conductance regulator. Biochemistry. 1997; 36(21):6455-61. DOI: 10.1021/bi970150v. View

10.

Weinreich F, Wood P, Riordan J, Nagel G . Direct action of genistein on CFTR. Pflugers Arch. 1997; 434(4):484-91. DOI: 10.1007/s004240050424. View

11.

Ashkenas J, Byers P . The final stage of gene expression: chaperones and the regulation of protein fate. Am J Hum Genet. 1997; 61(2):267-72. PMC: 1715893. DOI: 10.1086/514865. View

12.

Riordan J, Rommens J, Kerem B, Alon N, Rozmahel R, Grzelczak Z . Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989; 245(4922):1066-73. DOI: 10.1126/science.2475911. View

13.

Constantoulakis P, Knitter G, Stamatoyannopoulos G . On the induction of fetal hemoglobin by butyrates: in vivo and in vitro studies with sodium butyrate and comparison of combination treatments with 5-AzaC and AraC. Blood. 1989; 74(6):1963-71. View

14.

Rich D, Gregory R, Anderson M, Manavalan P, Smith A, Welsh M . Effect of deleting the R domain on CFTR-generated chloride channels. Science. 1991; 253(5016):205-7. DOI: 10.1126/science.1712985. View

15.

Hamosh A, Trapnell B, Zeitlin P, Montrose-Rafizadeh C, Rosenstein B, Crystal R . Severe deficiency of cystic fibrosis transmembrane conductance regulator messenger RNA carrying nonsense mutations R553X and W1316X in respiratory epithelial cells of patients with cystic fibrosis. J Clin Invest. 1991; 88(6):1880-5. PMC: 295756. DOI: 10.1172/JCI115510. View

16.

Dalemans W, Barbry P, Champigny G, Jallat S, Dott K, Dreyer D . Altered chloride ion channel kinetics associated with the delta F508 cystic fibrosis mutation. Nature. 1991; 354(6354):526-8. DOI: 10.1038/354526a0. View

17.

Knowles M, Clarke L, Boucher R . Extracellular ATP and UTP induce chloride secretion in nasal epithelia of cystic fibrosis patients and normal subjects in vivo. Chest. 1992; 101(3 Suppl):60S-63S. DOI: 10.1378/chest.101.3_supplement.60s. View

18.

Denning G, Anderson M, Amara J, Marshall J, Smith A, Welsh M . Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive. Nature. 1992; 358(6389):761-4. DOI: 10.1038/358761a0. View

19.

Yang Y, JANICH S, Cohn J, Wilson J . The common variant of cystic fibrosis transmembrane conductance regulator is recognized by hsp70 and degraded in a pre-Golgi nonlysosomal compartment. Proc Natl Acad Sci U S A. 1993; 90(20):9480-4. PMC: 47592. DOI: 10.1073/pnas.90.20.9480. View

20.

Ko Y, Thomas P, Delannoy M, Pedersen P . The cystic fibrosis transmembrane conductance regulator. Overexpression, purification, and characterization of wild type and delta F508 mutant forms of the first nucleotide binding fold in fusion with the maltose-binding protein. J Biol Chem. 1993; 268(32):24330-8. View