Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. - Wikidata - awgldk - TriplyDB

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

http://www.wikidata.org/entity/Q34483772

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Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications Advances and unmet needs in genetic, basic and clinical science in Alport syndrome: report from the 2015 International Workshop on Alport Syndrome New and emerging targeted therapies for cystic fibrosis Year in review 2015: Interstitial lung disease, pulmonary vascular disease, pulmonary function, sleep and ventilation, cystic fibrosis and paediatric lung disease Advancing clinical development pathways for new CFTR modulators in cystic fibrosis Targeted therapies to improve CFTR function in cystic fibrosis CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis Current and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its Symptoms Barriers to inhaled gene therapy of obstructive lung diseases: A review Pediatric Pulmonology year in review 2015: Part 4.Lipophosphoramidate-based bipolar amphiphiles: their syntheses and transfection properties.The UK National Neonatal Research Database: using neonatal data for research, quality improvement and more Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model.Evaluation of New Fluorescent Lipophosphoramidates for Gene Transfer and Biodistribution Studies after Systemic Administration A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer.Accurate reporting of adherence to inhaled therapies in adults with cystic fibrosis: methods to calculate "normative adherence".Cystic fibrosis: a model system for precision medicine.Lentiviral-mediated phenotypic correction of cystic fibrosis pigs.CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes Cystic fibrosis - a multiorgan protein misfolding disease.CFTR: A New Horizon in the Pathomechanism and Treatment of Pancreatitis.Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.Repairing the basic defect in cystic fibrosis - one approach is not enough.Tailored Pig Models for Preclinical Efficacy and Safety Testing of Targeted Therapies.Novel and Experimental Therapies in Chronic Pancreatitis.Cystic fibrosis: current therapeutic targets and future approaches.Current Progress in Therapeutic Gene Editing for Monogenic Diseases Strategies in early clinical development for the treatment of basic defects of cystic fibrosis.Evaluation of the transfection efficacies of quaternary ammonium salts prepared from sophorolipids.New drug developments in the management of cystic fibrosis lung disease.Genetic medicines for CF: Hype versus reality.Impact of gene editing on the study of cystic fibrosis.Inhaled gene delivery: a formulation and delivery approach.The story of cystic fibrosis 1965-2015.rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice.Cystic fibrosis: a clinical view.Gene Editing and Genetic Lung Disease. Basic Research Meets Therapeutic Application.Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems.

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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

http://www.wikidata.org/entity/Q34483772

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Alan W Maclean

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Alexandra L Quittner

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P2860

Dornase alfa for cystic fibrosis

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.

Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo.

Azithromycin maintenance therapy in patients with cystic fibrosis: a dose advice based on a review of pharmacokinetics, efficacy, and side effects.

Moving forward: cystic fibrosis gene therapy.

Delivery of genes into the CF airway.

Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung.

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.

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cystic fibrosis

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