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Cystic Fibrosis
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TLDR
Advances in understanding and treatment of cystic fibrosis are summarized, focusing on pulmonary disease, which accounts for most morbidity and deaths.Abstract:
Cystic fibrosis is the most common autosomal recessive disorder in white people, with a frequency of about 1 in 2500 livebirths. Discovery of the mutated gene encoding a defective chloride channel in epithelial cells--named cystic fibrosis transmembrane conductance regulator (CFTR)--has improved our understanding of the disorder's pathophysiology and has aided diagnosis, but has shown the disease's complexity. Gene replacement therapy is still far from being used in patients with cystic fibrosis, mostly because of difficulties of targeting the appropriate cells. Life expectancy of patients with the disorder has been greatly increased over past decades because of better notions of symptomatic treatment strategies. Here, we summarise advances in understanding and treatment of cystic fibrosis, focusing on pulmonary disease, which accounts for most morbidity and deaths.read more
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Nonantimicrobial Effects of Antibacterial Agents
Timothy R. Pasquale,James S. Tan +1 more
TL;DR: Some antimicrobials may have side effects that are not therapeutically useful, such as osmotic diuresis with high-dose beta -lactam administration, neuromuscular blockade of aminoglycosides, dysglycemia of fluoroquinolones, and serotonin syndrome with oxazolidinones.
Journal ArticleDOI
Chronic Lung Disease in Adolescents With Delayed Diagnosis of Vertically Acquired HIV Infection
Rashida A. Ferrand,Rashida A. Ferrand,Sujal R. Desai,Charlotte Hopkins,Caroline Elston,Susan J. Copley,Kusum Nathoo,Chiratidzo E. Ndhlovu,Shungu Munyati,Richard D. Barker,Robert F. Miller,Robert F. Miller,Tsitsi Bandason,Athol U. Wells,Elizabeth L. Corbett,Elizabeth L. Corbett,Elizabeth L. Corbett +16 more
TL;DR: A high burden of chronic lung disease was found among 116 consecutive adolescents with vertically acquired human immunodeficiency virus in Zimbabwe and the main cause of HIV-associated CLD appears to be obliterative bronchiolitis.
Journal ArticleDOI
Promoter hypomethylation of Toll-like receptor-2 gene is associated with increased proinflammatory response toward bacterial peptidoglycan in cystic fibrosis bronchial epithelial cells
Tsuyoshi Shuto,Takashi Furuta,Mariko Oba,Haidong Xu,Jian Dong Li,Judy Cheung,Dieter C. Gruenert,Dieter C. Gruenert,Dieter C. Gruenert,Akiko Uehara,Mary Ann Suico,Tsukasa Okiyoneda,Hirofumi Kai +12 more
TL;DR: It is shown that toll‐like receptor‐2 (TLR2) expression and response were strongly enhanced in the human CF bronchial epithelial cell line, CFBE41o‐, and suggested that TLR 2 overexpression or prolonged activation of TLR2 signaling might be critical in CF pathogenesis.
Journal ArticleDOI
Pharmacological properties of N-(3,5-diamino-6-chloropyrazine-2-carbonyl)-N'-4-[4-(2,3-dihydroxypropoxy)phenyl]butyl-guanidine methanesulfonate (552-02), a novel epithelial sodium channel blocker with potential clinical efficacy for cystic fibrosis lung disease.
Andrew J. Hirsh,Jim Zhang,Andra Zamurs,Jacquelyn Fleegle,William R. Thelin,Ray A. Caldwell,Juan R. Sabater,William M. Abraham,Mark Donowitz,Boyoung Cha,Kevin B. Johnson,Judith A. St. George,M. Ross Johnson,Richard C. Boucher +13 more
TL;DR: The preclinical data support the clinical use of 552-02 +/– hypertonic saline for CF lung disease and suggest a synergistic effect on ASL expansion was measured in canine or CF bronchial epithelia.
Journal ArticleDOI
Identification of N-acylhomoserine lactones in mucopurulent respiratory secretions from cystic fibrosis patients
TL;DR: A broad range of AHLs were detected and identified despite being present at low concentrations in limited sample volumes, and OHL and N-decanoyl-l-homoserine lactone were detected in nanomolar concentrations compared to picomolar amounts of the 3-oxo-derivatives of the AHLs identified.
References
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Journal ArticleDOI
Modifier genes in cystic fibrosis lung disease.
TL;DR: It is now clear that CFTR genotype alone does not account for the wide diversity in CF pulmonary phenotype and evidence is accumulating that secondary genetic factors separate from the CFTR locus significantly influence the severity of CF lung disease.
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