Diagnosis and treatment of cystic fibrosis in India: What is at stake for developing countries?

Baker MW, Atkins AE, Cordovado SK, et al. 2016 Improving newborn screening for cystic fibrosis using next-generation sequencing technology: a technical feasibility study. Genet. Med. 18 231–238

Article  CAS  PubMed  Google Scholar 

Bhakoo ON, Kumar R and Walia BNS 1968 Mucoviscidosis of the lung. Indian J. Pediatr. 35 183–185

Article  CAS  PubMed  Google Scholar 

Boeck KD, Munck A, Walker S, et al. 2014 Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J. Cyst. Fibros. 13 674–680

Article  PubMed  Google Scholar 

Bulcha JT, Wang Y, Ma H, et al. 2021 Viral vector platforms within the gene therapy landscape. Sig. Transduct. Target Ther. 6 1–24

Article  Google Scholar 

Carraro G, Perin L, Sedrakyan S, et al. 2008 Human amniotic fluid stem cells can integrate and differentiate into epithelial lung lineages. Stem Cells 26 2902–2911

Article  CAS  PubMed  Google Scholar 

Chamayou S, Sicali M, Lombardo D, et al. 2020 Universal strategy for preimplantation genetic testing for cystic fibrosis based on next generation sequencing. J. Assist. Reprod. Genet. 37 213–222

Article  PubMed  Google Scholar 

Clancy JP, Rowe SM, Accurso FJ, et al. 2012 Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax 67 12–18

Article  CAS  PubMed  Google Scholar 

Comeau AM, Accurso FJ, White TB, et al. 2007 Guidelines for implementation of cystic fibrosis newborn screening programs: Cystic Fibrosis Foundation Workshop Report. Pediatrics 119 495–518

Article  Google Scholar 

Condren ME and Bradshaw MD 2013 Ivacaftor: A novel gene-based therapeutic approach for cystic fibrosis. J. Pediatr. Pharmacol. Ther. 18 8–13

PubMed  PubMed Central  Google Scholar 

Cystic Fibrosis Foundation 2021 Annual Data Report (https://www.cff.org/sites/default/files/2021-11/Patient-Registry-Annual-Data-Report.pdf)

da Silva Filho LVRF, Zampoli M, Cohen-Cymberknoh M, et al. 2021 Cystic fibrosis in low and middle-income countries (LMIC): A view from four different regions of the world. Paediatr. Respir. Rev. 38 37–44

PubMed  Google Scholar 

Du M, Jones JR, Lanier J, et al. 2002 Aminoglycoside suppression of a premature stop mutation in a Cftr–/– mouse carrying a human CFTR-G542X transgene. J. Mol. Med. 80 595–604

Article  CAS  PubMed  Google Scholar 

Du M, Keeling KM, Fan L, et al. 2006 Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model. J. Mol. Med. 84 573–582

Article  CAS  PubMed  Google Scholar 

Farrell PM, Kosorok MR, Laxova A, et al. 1997 Nutritional benefits of neonatal screening for cystic fibrosis. Wisconsin Cystic Fibrosis Neonatal Screening Study Group. N. Engl. J. Med. 337 963–969

Article  CAS  PubMed  Google Scholar 

Farrell PM, Kosorok MR, Rock MJ, et al. 2001 Early diagnosis of cystic fibrosis through neonatal screening prevents severe malnutrition and improves long-term growth. Pediatrics 107 1–13

Article  CAS  PubMed  Google Scholar 

Farrow N, Miller D, Cmielewski P, et al. 2013 Airway gene transfer in a non-human primate: Lentiviral gene expression in marmoset lungs. Sci. Rep. 3 1287

Article  CAS  PubMed  PubMed Central  Google Scholar 

Flotte TR, Zeitlin PL, Reynolds TC, et al. 2003 Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum. Gene Ther. 14 1079–1088

Article  CAS  PubMed  Google Scholar 

Flotte TR, Ng P, Dylla DE, et al. 2007 Viral vector–mediated and cell-based therapies for treatment of cystic fibrosis. Mol. Ther. 15 229–241

Article  CAS  PubMed  Google Scholar 

Flume PA, Liou TG, Borowitz DS, et al. 2012 Ivacaftor in subjects with cystic fibrosis who are homozygous for the f508del-CFTR mutation. CHEST 142 718–724

Article  PubMed  PubMed Central  Google Scholar 

Ghosh S, Brown AM, Jenkins C, et al. 2020 Viral Vector systems for gene therapy: a comprehensive literature review of progress and biosafety challenges. Appl. Biosaf. 25 7–18

Article  PubMed  PubMed Central  Google Scholar 

Guo J, Garratt A and Hill A 2022a Worldwide rates of diagnosis and effective treatment for cystic fibrosis. J. Cyst. Fibros. 21 456–462

Article  PubMed  Google Scholar 

Guo J, Wang J, Zhang J, et al. 2022b Current prices versus minimum costs of production for CFTR modulators. J. Cyst. Fibros. 21 866–872

Article  CAS  PubMed  Google Scholar 

Hida K, Lai SK, Suk JS, et al. 2011 Common gene therapy viral vectors do not efficiently penetrate sputum from cystic fibrosis patients. PLoS One 6 19919

Article  Google Scholar 

Jat KR 2013 Spirometry in children. Prim. Care. Respir. J 22 221–229

Article  PubMed  PubMed Central  Google Scholar 

Johnson C, Butler SM, Konstan MW, et al. 2003 Factors influencing outcomes in cystic fibrosis: a center-based analysis. CHEST 123 20–27

Article  PubMed  Google Scholar 

Kabra SK, Kabra M, Connett GJ, et al. 1999 Diagnosis of cystic fibrosis: Indian perspective. Indian J. Pediatr. 66 923–928

Article  CAS  PubMed  Google Scholar 

Kabra M, Kabra SK, Ghosh M, et al. 2000 Is the spectrum of mutations in Indian patients with cystic fibrosis different? Am. J. Med. Genet. 93 161–163

Article  CAS  PubMed  Google Scholar 

Kabra SK, Kabra M, Gera S, et al. 2002 An indigenously developed method for sweat collection and estimation of chloride for diagnosis of cystic fibrosis. Indian Pediatr. 39 1039–1043

CAS  PubMed  Google Scholar 

Kabra SK, Kabra M, Lodha R, et al. 2003 Clinical profile and frequency of Delta F508 mutation in Indian children with cystic fibrosis. Indian Pediatr. 40 612–619

CAS  PubMed  Google Scholar 

Kabra SK, Kabra M, Lodha R, et al. 2007 Cystic fibrosis in India. Pediatr. Pulmonol. 42 1087–1094

Article  CAS  PubMed  Google Scholar 

Kerem E, Konstan MW, Boeck KD, et al. 2014 Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. Lancet Respir. Med. 2 539–547

Article  CAS  PubMed  Google Scholar 

Kim N, Duncan GA, Hanes J, et al. 2016 Barriers to inhaled gene therapy of obstructive lung diseases: A review. J. Control. Release. 240 465–488

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kiseleva A, Klimushina M, Sotnikova E, et al. 2020 Cystic fibrosis polymorphic variants in a Russian population. Pharmgenomics Pers. Med. 13 679–686

CAS  PubMed  PubMed Central  Google Scholar 

Konstan MW, Butler SM, Wohl MEB, et al. 2003 Growth and nutritional indexes in early life predict pulmonary function in cystic fibrosis. J. Pediatr. 142 624–630

Article  PubMed 

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