Pulmonary delivery of sirna for the treatment of cystic fibrosis and pulmonary delivery platforms
Cystic fibrosis (CF) is one of the most deadly diseases of lungs that involves symptoms such as breathing difficulties, coughing and lung infection. Despite important therapeutic advances, the definitive treatment for CF remains elusive. CF is a good candidate for gene therapy because it is relatively common, lethal and monogenic and it does not have adequate treatment options. In this review article, we have reviewed gene therapy as a potential treatment option for CF. Various platforms and strategies for pulmonary gene delivery have also been discussed in detail.
2. Rommens JM, Iannuzzi MC, Kerem B-s, Drumm ML, Melmer G, Dean M, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989;245(4922):1059-65.
3. Zaman MK, Milani DN, Bhatt P, Hariyadi DM, Athiyah U, Islam J, et al. Nutraceuticals: Formulations and Commercialization. Kirk‐Othmer Encyclopedia of Chemical Technology. p. 1-29.
4. Wagner JA, Gardner P. Toward cystic fibrosis gene therapy. Annual review of medicine. 1997;48:203-16.
5. Stutts MJ, Canessa CM, Olsen JC, Hamrick M. CFTR as a cAMP-dependent regulator of sodium channels. Science. 1995;269(5225):847.
6. Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell. 1993;73(7):1251-4.
7. Dalemans W, Barbry P, Champigny G, Jallat S, Jallat S, Dott K, et al. Altered chloride ion channel kinetics associated with the ΔF508 cystic fibrosis mutation. Nature. 1991;354(6354):526-8.
8. Bhatt P, Narvekar P. Challenges and Strategies for Drug Transport across the Blood Brain Barrier. 2018;3:17-21.
9. Smith JJ, Travis SM, Greenberg EP, Welsh MJ. Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell. 1996;85(2):229-36.
10. Pier GB, Grout M, Zaidi TS, Olsen JC, Johnson LG, Yankaskas JR, et al. Role of mutant CFTR in hypersusceptibility of cystic fibrosis patients to lung infections. Science (New York, NY). 1996;271(5245):64.
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