Cystic fibrosis precision therapeutics: emerging considerations
D Joshi, A Ehrhardt, JS Hong… - Pediatric …, 2019 - Wiley Online Library
… ” interventions for specific abnormalities of the cystic fibrosis … with CF worldwide—barriers
to precision therapy remain. These … has occurred regarding CF precision medicine, this review …
to precision therapy remain. These … has occurred regarding CF precision medicine, this review …
Cystic fibrosis–an example of personalized and precision medicine
M Skov, CR Hansen, T Pressler - Apmis, 2019 - Wiley Online Library
… Cystic fibrosis (CF) is a severe, monogenic, autosomal recessive disease caused by
mutations in the CFTR (cystic fibrosis transmembrane regulator) gene, where disturbed chloride …
mutations in the CFTR (cystic fibrosis transmembrane regulator) gene, where disturbed chloride …
CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine
M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
… Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF … Mutations
in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (…
in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (…
Making precision medicine personal for cystic fibrosis
C Manfredi, JM Tindall, JS Hong, EJ Sorscher - Science, 2019 - science.org
… It was originally anticipated that well-established disease subclasses would serve as an
organizing principle for precision CF treatments and help identify specific compounds for …
organizing principle for precision CF treatments and help identify specific compounds for …
Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy
DH Tewkesbury, RC Robey, PJ Barry - Breathe, 2021 - Eur Respiratory Soc
… The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in … , treatment
advances had focused on the downstream consequences of a dysfunctional cystic fibrosis …
advances had focused on the downstream consequences of a dysfunctional cystic fibrosis …
Correlating cystic fibrosis transmembrane conductance regulator function with clinical features to inform precision treatment of cystic fibrosis
AF McCague, KS Raraigh, MJ Pellicore… - American journal of …, 2019 - atsjournals.org
… of precision treatment for cystic fibrosis using small-molecule therapeutics has created a
need to estimate potential clinical improvements attributable to increases in cystic fibrosis …
need to estimate potential clinical improvements attributable to increases in cystic fibrosis …
Precision medicine based on CFTR genotype for people with cystic fibrosis
I Haq, M Almulhem, S Soars, D Poulton… - Pharmacogenomics …, 2022 - Taylor & Francis
… cystic fibrosis transmembrane conductance regulator gene. This causes multisystem disease
due to dysfunction of the cystic fibrosis … Until recently, treatment was directed at managing …
due to dysfunction of the cystic fibrosis … Until recently, treatment was directed at managing …
Cystic fibrosis lung disease modifiers and their relevance in the new era of precision medicine
A Sepahzad, DJ Morris-Rosendahl, JC Davies - Genes, 2021 - mdpi.com
… Our understanding of cystic fibrosis (CF) has grown exponentially since the discovery of
the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989. With evolving …
the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989. With evolving …
The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
… with cystic fibrosis who are genetically eligible for treatment, … the pathogenesis of cystic
fibrosis, working together with a … of the lungs of people with cystic fibrosis. We review progress in (…
fibrosis, working together with a … of the lungs of people with cystic fibrosis. We review progress in (…
The influence of CFTR complex alleles on precision therapy of cystic fibrosis
B Chevalier, A Hinzpeter - Journal of Cystic Fibrosis, 2020 - Elsevier
… propose adequate treatments. Conversely, cis-variants can affect treatment response when
… We wish to thank the French cystic fibrosis association Vaincre la mucoviscidose for funding …
… We wish to thank the French cystic fibrosis association Vaincre la mucoviscidose for funding …
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