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

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, +77

The Lancet Respiratory Medicine

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

Eric W.F.W. Alton, Jeffrey M. Beekman, A. Christopher Boyd, +35

Thorax

Translational MedicineLung Gene TransferViral DiagnosticsViral PathogenesisPharmacology +13

Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP.

Gerry McLachlan, Ling‐Pei Ho, Hazel Davidson‐Smith, +15

PubMed

Differential global gene expression in cystic fibrosis nasal and bronchial epithelium

Varrie Ogilvie, Margaret R. Passmore, Laura Hyndman, +11

Genomics

FibrosisCystic Fibrosis NasalAllergyPulmonary FibrosisBronchial Epithelium +4

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby, +77

Efficacy and Mechanism Evaluation

Secondary Clinical OutcomesInflammatory Lung DiseaseIn Vivo Gene TherapyLung InflammationAdvanced Lung Disease +14

Chimeric constructs endow the human CFTR Cl ⁻ channel with the gating behavior of murine CFTR

Toby S. Scott‐Ward, Zhiwei Cai, Elizabeth S. Dawson, +8

Proceedings of the National Academy of Sciences

Physiological ResearchSignal TransductionMolecular PhysiologyNeurophysiologyNatural Sciences +13

The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep

Eric W.F.W. Alton, Alison Baker, Eilidh Baker, +23

Biomaterials

Animal PhysiologyInflammationAllergyRespiratory ToxicologyPathogenesis +4

Insertion of natural intron 6a‐6b into a human cDNA‐derived gene therapy vector for cystic fibrosis improves plasmid stability and permits facile RNA/DNA discrimination

A. Christopher Boyd, Friedrich Popp, Uwe Michaelis, +6

The Journal of Gene Medicine

Retroviral transduction of enriched hematopoietic stem cells allows lifelong Bcl-2 expression in multiple lineages but does not perturb hematopoiesis

Karin M Innes, Stephen J. Szilvassy, Heather Davidson, +3

Experimental Hematology

Multiple LineagesRetroviral TransductionMedicineStem Cell MobilizationImmunology +9

An immunocytochemical assay to detect human CFTR expression following gene transfer

Heather Davidson, Abigail Wilson, Robert D. Gray, +21

Molecular and Cellular Probes

Signal TransductionMolecular PhysiologyProtein ExpressionReporter Gene AssayImmunology +7