Literature DB >> 26623687

A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial.

Eric W F W Alton1,2, A Christopher Boyd3, David J Porteous3, Gwyneth Davies1,2, Jane C Davies1,2, Uta Griesenbach1,2, Tracy E Higgins1,2, Deborah R Gill4, Stephen C Hyde4, J Alastair Innes5.   

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Year:  2015        PMID： 26623687      PMCID： PMC4731704          DOI： 10.1164/rccm.201506-1193LE

Source DB:  PubMed          Journal:  Am J Respir Crit Care Med        ISSN： 1073-449X            Impact factor:   21.405


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  9 in total

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

Authors:  G McLachlan; H Davidson; E Holder; L A Davies; I A Pringle; S G Sumner-Jones; A Baker; P Tennant; C Gordon; C Vrettou; R Blundell; L Hyndman; B Stevenson; A Wilson; A Doherty; D J Shaw; R L Coles; H Painter; S H Cheng; R K Scheule; J C Davies; J A Innes; S C Hyde; U Griesenbach; E W F W Alton; A C Boyd; D J Porteous; D R Gill; D D S Collie
Journal:  Gene Ther       Date:  2011-04-21       Impact factor: 5.250

Review 2.  Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.

Authors:  Uta Griesenbach; Eric W F W Alton
Journal:  Adv Drug Deliv Rev       Date:  2008-12-24       Impact factor: 15.470

Review 3.  CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.

Authors:  Stephen C Hyde; Ian A Pringle; Syahril Abdullah; Anna E Lawton; Lee A Davies; Anusha Varathalingam; Graciela Nunez-Alonso; Anne-Marie Green; Reto P Bazzani; Stephanie G Sumner-Jones; Mario Chan; Hongyu Li; Nelson S Yew; Seng H Cheng; A Christopher Boyd; Jane C Davies; Uta Griesenbach; David J Porteous; David N Sheppard; Felix M Munkonge; Eric W F W Alton; Deborah R Gill
Journal:  Nat Biotechnol       Date:  2008-04-27       Impact factor: 54.908

4.  Aerosol delivery of DNA/liposomes to the lung for cystic fibrosis gene therapy.

Authors:  Lee A Davies; Graciela A Nunez-Alonso; Gerry McLachlan; Stephen C Hyde; Deborah R Gill
Journal:  Hum Gene Ther Clin Dev       Date:  2014-05-27       Impact factor: 5.032

5.  A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.

Authors:  Bonnie W Ramsey; Jane Davies; N Gerard McElvaney; Elizabeth Tullis; Scott C Bell; Pavel Dřevínek; Matthias Griese; Edward F McKone; Claire E Wainwright; Michael W Konstan; Richard Moss; Felix Ratjen; Isabelle Sermet-Gaudelus; Steven M Rowe; Qunming Dong; Sally Rodriguez; Karl Yen; Claudia Ordoñez; J Stuart Elborn
Journal:  N Engl J Med       Date:  2011-11-03       Impact factor: 91.245

Review 6.  Lung clearance index: evidence for use in clinical trials in cystic fibrosis.

Authors:  L Kent; P Reix; J A Innes; S Zielen; M Le Bourgeois; C Braggion; S Lever; H G M Arets; K Brownlee; J M Bradley; K Bayfield; K O'Neill; D Savi; D Bilton; A Lindblad; J C Davies; I Sermet; K De Boeck
Journal:  J Cyst Fibros       Date:  2013-12-05       Impact factor: 5.482

7.  Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter.

Authors:  D R Gill; S E Smyth; C A Goddard; I A Pringle; C F Higgins; W H Colledge; S C Hyde
Journal:  Gene Ther       Date:  2001-10       Impact factor: 5.250

8.  Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways.

Authors:  A C Rose; C A Goddard; W H Colledge; S H Cheng; D R Gill; S C Hyde
Journal:  Gene Ther       Date:  2002-10       Impact factor: 5.250

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

Authors:  Eric W F W Alton; David K Armstrong; Deborah Ashby; Katie J Bayfield; Diana Bilton; Emily V Bloomfield; A Christopher Boyd; June Brand; Ruaridh Buchan; Roberto Calcedo; Paula Carvelli; Mario Chan; Seng H Cheng; D David S Collie; Steve Cunningham; Heather E Davidson; Gwyneth Davies; Jane C Davies; Lee A Davies; Maria H Dewar; Ann Doherty; Jackie Donovan; Natalie S Dwyer; Hala I Elgmati; Rosanna F Featherstone; Jemyr Gavino; Sabrina Gea-Sorli; Duncan M Geddes; James S R Gibson; Deborah R Gill; Andrew P Greening; Uta Griesenbach; David M Hansell; Katharine Harman; Tracy E Higgins; Samantha L Hodges; Stephen C Hyde; Laura Hyndman; J Alastair Innes; Joseph Jacob; Nancy Jones; Brian F Keogh; Maria P Limberis; Paul Lloyd-Evans; Alan W Maclean; Michelle C Manvell; Dominique McCormick; Michael McGovern; Gerry McLachlan; Cuixiang Meng; M Angeles Montero; Hazel Milligan; Laura J Moyce; Gordon D Murray; Andrew G Nicholson; Tina Osadolor; Javier Parra-Leiton; David J Porteous; Ian A Pringle; Emma K Punch; Kamila M Pytel; Alexandra L Quittner; Gina Rivellini; Clare J Saunders; Ronald K Scheule; Sarah Sheard; Nicholas J Simmonds; Keith Smith; Stephen N Smith; Najwa Soussi; Samia Soussi; Emma J Spearing; Barbara J Stevenson; Stephanie G Sumner-Jones; Minna Turkkila; Rosa P Ureta; Michael D Waller; Marguerite Y Wasowicz; James M Wilson; Paul Wolstenholme-Hogg
Journal:  Lancet Respir Med       Date:  2015-07-03       Impact factor: 30.700
  9 in total
  15 in total

1.  Treating Cystic Fibrosis with mRNA and CRISPR.

Authors:  Alejandro Da Silva Sanchez; Kalina Paunovska; Ana Cristian; James E Dahlman
Journal:  Hum Gene Ther       Date:  2020-09-08       Impact factor: 5.695

Review 2.  Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF.

Authors:  Marie E Egan
Journal:  Pediatr Pulmonol       Date:  2021-02

3.  Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis.

Authors:  Ema Robinson; Kelvin D MacDonald; Kai Slaughter; Madison McKinney; Siddharth Patel; Conroy Sun; Gaurav Sahay
Journal:  Mol Ther       Date:  2018-06-15       Impact factor: 11.454

4.  A multiple reader scoring system for Nasal Potential Difference parameters.

Authors:  George M Solomon; Bo Liu; Isabelle Sermet-Gaudelus; Isabelle Fajac; Michael Wilschanski; Francois Vermeulen; Steven M Rowe
Journal:  J Cyst Fibros       Date:  2017-04-29       Impact factor: 5.482

5.  Lentiviral-mediated phenotypic correction of cystic fibrosis pigs.

Authors:  Ashley L Cooney; Mahmoud H Abou Alaiwa; Viral S Shah; Drake C Bouzek; Mallory R Stroik; Linda S Powers; Nick D Gansemer; David K Meyerholz; Michael J Welsh; David A Stoltz; Patrick L Sinn; Paul B McCray
Journal:  JCI Insight       Date:  2016-09-08

Review 6.  Cystic fibrosis: a model system for precision medicine.

Authors:  Stacey L Martiniano; Scott D Sagel; Edith T Zemanick
Journal:  Curr Opin Pediatr       Date:  2016-06       Impact factor: 2.856

Review 7.  Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.

Authors:  Elena K Schneider-Futschik
Journal:  Gene Ther       Date:  2019-07-12       Impact factor: 5.250

Review 8.  Methods for CRISPR-Cas as Ribonucleoprotein Complex Delivery In Vivo.

Authors:  Alesya G Bykonya; Alexander V Lavrov; Svetlana A Smirnikhina
Journal:  Mol Biotechnol       Date:  2022-03-24       Impact factor: 2.695

Review 9.  Strategies for the etiological therapy of cystic fibrosis.

Authors:  Luigi Maiuri; Valeria Raia; Guido Kroemer
Journal:  Cell Death Differ       Date:  2017-09-22       Impact factor: 15.828

Review 10.  Liposome Delivery Systems for Inhalation: A Critical Review Highlighting Formulation Issues and Anticancer Applications.

Authors:  Mindaugas Rudokas; Mohammad Najlah; Mohamed Albed Alhnan; Abdelbary Elhissi
Journal:  Med Princ Pract       Date:  2016-03-02       Impact factor: 1.927
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