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2021 ◽  
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Ofra Avizur-Barchad ◽  
Efrat Ozeri-Galai ◽  
Renana Elgrabli ◽  
Meital R. Schirelman ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Nonsense Mutation ◽  
Antisense Oligonucleotide ◽  
Therapeutic Approach

scholarly journals Small molecule‐facilitated anion transporters in cells for a novel therapeutic approach to cystic fibrosis

2019 ◽  
Vol 176 (11) ◽  
pp. 1764-1779 ◽  
Author(s):  
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Claudia Cossu ◽  
Valeria Capurro ◽  
Cristiana Picco ◽  
Alessandra Ludovico ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Small Molecule ◽  
Therapeutic Approach ◽  
Anion Transporters ◽  
Novel Therapeutic ◽  
In Cells

Ataluren in nonsense mutation cystic fibrosis patients not receiving chronic inhaled tobramycin: Evaluation of exacerbations and lung function

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Isabelle Sermet-Gaudelus ◽  
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Anne Malfroot ◽  
Harry Heijerman ◽  
Eitan Kerem ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Nonsense Mutation

scholarly journals Nine cystic fibrosis patients homozygous for the CFTR nonsense mutation R1162X have mild or moderate lung disease.

1992 ◽  
Vol 29 (8) ◽  
pp. 558-562 ◽  
Author(s):  
P Gasparini ◽  
G Borgo ◽  
G Mastella ◽  
A Bonizzato ◽  
M Dognini ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Nonsense Mutation

scholarly journals Inhaled ENaC antisense oligonucleotide ameliorates cystic fibrosis-like lung disease in mice

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pp. 671-680 ◽  
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Chenguang Zhao ◽  
Chong Jiang ◽  
Dong Bai ◽  
Melanie Katz ◽  
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Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Antisense Oligonucleotide

scholarly journals 661: W1282X cystic fibrosis mouse models for examining in vivo nonsense mutation correction

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pp. S313-S314
Author(s):  
M. Michicich ◽  
Z. Traylor ◽  
D. LePage ◽  
R. Mann ◽  
W. Jiang ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Mouse Models ◽  
Nonsense Mutation ◽  
Cystic Fibrosis Mouse
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