Abo Bibliothek: Guest
Digitales Portal Digitale Bibliothek eBooks Zeitschriften Referenzen und Berichte Forschungssammlungen
Critical Reviews™ in Eukaryotic Gene Expression
Impact-faktor: 2.156 5-jähriger Impact-Faktor: 2.255 SJR: 0.649 SNIP: 0.599 CiteScore™: 3

ISSN Druckformat: 1045-4403
ISSN Online: 2162-6502

Critical Reviews™ in Eukaryotic Gene Expression

DOI: 10.1615/CritRevEukaryotGeneExpr.2020030845
pages 191-197

Pharmacological and Genomic Approaches in Management of Cystic Fibrosis

Muhammad Imran Qadir
Institute of Molecular Biology and Biotechnology, Bahauddin Zakariya University, Multan, Pakistan
Iqra Ali Yameen
Institute of Molecular Biology and Biotechnology, Bahauddin Zakariya University, Multan, Pakistan

ABSTRAKT

Cystic fibrosis (CF) is an inherited recessive autosomal disorder that affects the lungs, the digestive system, and secretory glands. It is a lethal condition caused by a mutation in the gene cystic-fibrosis-transmembrane-conductance- regulator (CFTR), which leads to defects in ion channels and results in obstruction of mucus in airway channels. Unbalanced ion exchange causes impaired water transport and accumulation of viscous mucus in the air way leads to bacterial colonization, for example, with Staphylococcus aureus. The most common mutation is the deletion of nucleotides in epithelial membrane; hence, it is a multiple-organ−defective disease that mostly effects the lungs. Researchers are working on gene therapy that aims to introduce a normal CFTR gene copy into the epithelial cells of lungs. Several approaches have been designed to improve transepithelial ion transport in CF patients. Normal CFTR gene delivery has been performed using viral and nonviral vectors, but these approaches are not more efficient against the cell barriers. Enzymes may be used that inhibit the sphingolipid to provide proper microenvironment for the CFTR gene product. Thymosin alpha-1 has also been reported as a potential corrector in treatment of CF.

REFERENZEN

  1. Ratjen FA. Cystic fibrosis: Pathogenesis and future treatment strategies. Respir Care. 2009;54(5):595-605.

  2. Verrecchia F, Mauviel A. Transforming growth factor-beta and fibrosis. World J Gastroenterol. 2007;13(22):3056-62.

  3. Cebotaru L, Rapino D, Cebotaru V, Guggino WB. Correcting the cystic fibrosis disease mutant, A455E CFTR. PLoS One. 2014;9(1):e85183.

  4. Bookout AL, Mangelsdorf DJ. Quantitative real-time PCR protocol for analysis of nuclear receptor signaling pathways. Nucl Recept Signal. 2003;1(1):e012.

  5. Riordan JR, Rommens JM, Kerem B-S, Alon N, Rozmahel R, Grzelczak Z, Zielenski J, Lok S, Plavsic N, Chou J-L. Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA. Science. 1989;245(4922):1066-73.

  6. Qadir MI, Mushtaq H, Mobeen T. In-silico study of potential carboxylic acid derivatives as D-glutamate ligase inhibitors in Salmonella typhi. Kuwait J Sci. 2018;45(1):100-60.

  7. Qadir MI. Qadir theory of cancer etiology. Crit Rev Eukar Gene Expr. 2018;28(1):13-15.

  8. Qadir MI. Hepatitis in AIDS patients. Rev Med Virol. 2018;28(1):e1956.

  9. Qadir MI, Chauhdary Z. Antibacterial activity of novel strains of bactiophages: An experimental approach. Crit Rev Eukar Gene Expr. 2018;28(1):1-12.

  10. Qadir MI, Manzoor A, Akash MSH. Potential role of medicinal plants for anti-atherosclerosis activity. Bangladesh J Pharma. 2018;13(1):59-66.

  11. Glass M, inventor; Syngenta Ltd., assignee. Treatment of cystic fibrosis. United States Patent US5162348A. 1992 Nov 11.

  12. Robbins PD, Ghivizzani SC. Viral vectors for gene therapy. Pharmacol Therapeut. 1998;80(1):35-47.

  13. Frederiksen B, Koch C, Hoiby N. Antibiotic treatment of initial colonization with Pseudomonas aeruginosa postpones chronic infection and prevents deterioration of pulmonary function in cystic fibrosis. Pediatr Pulm. 1997;23(5):330-35.

  14. Hoiby N. Recent advances in the treatment of Pseudomonas aeruginosa infections in cystic fibrosis. BMC Med. 2011;9(1):32.

  15. Savla R, Minko T. Nanotechnology approaches for inhalation treatment of fibrosis. J Drug Target. 2013;21(10):914-25.

  16. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-72.

  17. Konstan MW, Byard PJ, Hoppel CL, Davis PB. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med. 1995;332(13):848-54.

  18. Aerts JMFG, Boot RG, inventors; Academisch Medisch Centrum, assignee. Treatment of cystic fibrosis. United States Patent 8410081B2. 2013 Apr 4.

  19. Norez C, Noel S, Wilke M, Bijvelds M, Jorna H, Melin P, DeJonge H, Becq F. Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the α-glucosidase inhibitor miglustat. FEBS Lett. 2006;580(8):2081-86.

  20. Boot RG, Verhoek M, Donker-Koopman W, Strijland A, van Marle J, Overkleeft HS, Wennekes T, Aerts JM. Identification of the non-lysosomal glucosylceramidase as β-glucosidase 2. J Biol Chem. 2007;282(2):1305-12.

  21. Overkleeft HS, Renkema GH, Neele J, Vianello P, Hung IO, Strijland A, Van Der Burg AM, Koomen G-J, Pandit UK, Aerts JM. Generation of specific deoxynojirimycin-type inhibitors of the non-lysosomal glucosylceramidase. J Biol Chem. 1998;273(41):26522-27.

  22. Belcher C, Vij N. Protein processing and inflammatory signaling in cystic fibrosis: Challenges and therapeutic strategies. Curr Mol Med. 2010;10(1):82-94.

  23. Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med. 2015;373(3):220-31.

  24. Cholon DM, Quinney NL, Fulcher ML, Esther CR, Das J, Dokholyan NV, Randell SH, Boucher RC, Gentzsch M. Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis. Sci Transl Med. 2014;6(246):246ra296.

  25. Conrad C, Lymp J, Thompson V, Dunn C, Davies Z, Chatfield B, Nichols D, Clancy J, Vender R, Egan M. Long-term treatment with oral N-acetylcysteine: Affects lung function but not sputum inflammation in cystic fibrosis subjects. A phase II randomized placebo-controlled trial. J Cyst Fibros. 2015;14(2):219-27.

  26. Goldstein AL, Guha A, Zatz MM, Hardy MA, White A. Purification and biological activity of thymosin, a hormone of the thymus gland. Proc Natl Acad Sci. 1972;69(7):1800-3.

  27. Romani L, Bistoni F, Perruccio K, Montagnoli C, Gaziano R, Bozza S, Bonifazi P, Bistoni G, Rasi G, Velardi A. Thymosin α1 activates dendritic cell tryptophan catabolism and establishes a regulatory environment for balance of inflammation and tolerance. Blood. 2006;108(7):2265-74.

  28. Romani L, Oikonomou V, Moretti S, Iannitti RG, D'Adamo MC, Villella VR, Pariano M, Sforna L, Borghi M, Bellet MM, Fallarino F, Pallotta MT, Servillo G, Ferrari E, Puccetti P, Kroemer G, Pessia M, Maiuri L, Goldstein AL, Garaci E. Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis. Nat Med. 2017;23(5):590-600.

  29. Romani L, Garaci E, inventors; Sciclone Pharmaceuticals International Ltd., assignee. Thymosin alpha 1 for use in treatment of cystic fibrosis. United States Patent US005162348. 2019 Nov 11.


Articles with similar content:

The Role of Homologous Recombination and Non-Homologous Ends Joining Systems in M. Tuberculosis Survival inside Macrophages
Onco Therapeutics, Vol.6, 2015, issue 3-4
Michal Kielbik, Magdalena Klink, Izabela Szulc-Kielbik
The Global Problem of Antibiotic Resistance
Critical Reviews™ in Immunology, Vol.30, 2010, issue 1
Thomas D. Gootz
Genetically Modified Aedes aegypti to Control Dengue: A Review
Critical Reviews™ in Eukaryotic Gene Expression, Vol.27, 2017, issue 4
Muhammad Masoud, Muhammad Qsim, Ijaz Rasul, Namrah Noor, Usman Ali Ashfaq, Muhammad Zubair Yousaf, Azfar Hussain
Drug Targeting by Surface Cationization
Critical Reviews™ in Therapeutic Drug Carrier Systems, Vol.17, 2000, issue 5
Susan Moody Haupt, Tareq Taha Jubeh, Abraham Rubinstein, Sigal Blau
The Route to HPV-Associated Neoplastic Transformation: A Review of the Literature
Critical Reviews™ in Eukaryotic Gene Expression, Vol.26, 2016, issue 1
Nedime Serakinci, Pinar Tulay