Literature DB >> 23525080

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis.

Eric W F W Alton1, A Christopher Boyd, Seng H Cheng, Steve Cunningham, Jane C Davies, Deborah R Gill, Uta Griesenbach, Tracy Higgins, Stephen C Hyde, J Alastair Innes, Gordon D Murray, David J Porteous.   

Abstract

The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a non-viral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.

Entities:  

Keywords:  Cystic Fibrosis

Mesh：

Substances：

Year:  2013        PMID： 23525080     DOI： 10.1136/thoraxjnl-2013-203309

Source DB:  PubMed          Journal:  Thorax        ISSN： 0040-6376            Impact factor:   9.139


  20 in total

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2.  Multiple forms of casein kinase from rabbit erythrocytes.

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Journal:  Biochim Biophys Acta       Date:  1975-11-20

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Review 5.  New and Emerging Treatments for Cystic Fibrosis.

Authors:  Peter J Barry; Andrew M Jones
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6.  Efficient intratracheal delivery of airway epithelial cells in mice and pigs.

Authors:  Liqiong Gui; Hong Qian; Kevin A Rocco; Loreta Grecu; Laura E Niklason
Journal:  Am J Physiol Lung Cell Mol Physiol       Date:  2015-01-15       Impact factor: 5.464

Review 7.  Ferret and pig models of cystic fibrosis: prospects and promise for gene therapy.

Authors:  Ziying Yan; Zoe A Stewart; Patrick L Sinn; John C Olsen; Jim Hu; Paul B McCray; John F Engelhardt
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Review 8.  Cystic fibrosis.

Authors:  Felix Ratjen; Scott C Bell; Steven M Rowe; Christopher H Goss; Alexandra L Quittner; Andrew Bush
Journal:  Nat Rev Dis Primers       Date:  2015-05-14       Impact factor: 52.329

Review 9.  Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease.

Authors:  Tim W R Lee; Kevin W Southern; Luke A Perry; Jahan C Penny-Dimri; Aisha A Aslam
Journal:  Cochrane Database Syst Rev       Date:  2016-06-17

Review 10.  Gene and cell therapy for children--new medicines, new challenges?

Authors:  Karen F Buckland; H Bobby Gaspar
Journal:  Adv Drug Deliv Rev       Date:  2014-02-28       Impact factor: 15.470
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