Literature DB >> 9659356

Gene therapy for cystic fibrosis: which postman, which box?

P G Middleton1, E W Alton.   

Abstract

Since 1989 when the gene responsible for cystic fibrosis was cloned and designated the cystic fibrosis transmembrane conductance regulator (CFTR) gene, considerable progress has been made in the development of gene therapy for this disease. Clinical trials have already been performed using cationic liposome and adenoviral based gene transfer systems, measuring the safety and efficacy of this new form of treatment, with variable results to date. These two approaches and the current progress in airway gene delivery are discussed.

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Year:  1998        PMID: 9659356      PMCID: PMC1745161          DOI: 10.1136/thx.53.3.197

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


  12 in total

1.  Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis.

Authors:  R G Crystal; N G McElvaney; M A Rosenfeld; C S Chu; A Mastrangeli; J G Hay; S L Brody; H A Jaffe; N T Eissa; C Danel
Journal:  Nat Genet       Date:  1994-09       Impact factor: 38.330

2.  A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction.

Authors:  J R Dorin; R Farley; S Webb; S N Smith; E Farini; S J Delaney; B J Wainwright; E W Alton; D J Porteous
Journal:  Gene Ther       Date:  1996-09       Impact factor: 5.250

3.  Mode of formation and structural features of DNA-cationic liposome complexes used for transfection.

Authors:  H Gershon; R Ghirlando; S B Guttman; A Minsky
Journal:  Biochemistry       Date:  1993-07-20       Impact factor: 3.162

4.  Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium.

Authors:  P G Middleton; D M Geddes; E W Alton
Journal:  Eur Respir J       Date:  1994-11       Impact factor: 16.671

5.  Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice.

Authors:  E W Alton; P G Middleton; N J Caplen; S N Smith; D M Steel; F M Munkonge; P K Jeffery; D M Geddes; S L Hart; R Williamson
Journal:  Nat Genet       Date:  1993-10       Impact factor: 38.330

6.  Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.

Authors:  J Zabner; L A Couture; R J Gregory; S M Graham; A E Smith; M J Welsh
Journal:  Cell       Date:  1993-10-22       Impact factor: 41.582

7.  Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis.

Authors:  L G Johnson; J C Olsen; B Sarkadi; K L Moore; R Swanstrom; R C Boucher
Journal:  Nat Genet       Date:  1992-09       Impact factor: 38.330

8.  Nasal application of the cationic liposome DC-Chol:DOPE does not alter ion transport, lung function or bacterial growth.

Authors:  P G Middleton; N J Caplen; X Gao; L Huang; H Gaya; D M Geddes; E W Alton
Journal:  Eur Respir J       Date:  1994-03       Impact factor: 16.671

9.  Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor.

Authors:  T Ferkol; J C Perales; E Eckman; C S Kaetzel; R W Hanson; P B Davis
Journal:  J Clin Invest       Date:  1995-02       Impact factor: 14.808

10.  Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis.

Authors:  Y Yang; F A Nunes; K Berencsi; E Gönczöl; J F Engelhardt; J M Wilson
Journal:  Nat Genet       Date:  1994-07       Impact factor: 38.330

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  5 in total

Review 1.  Genetic targeting of the renin-angiotensin system for long-term control of hypertension.

Authors:  Beverly L Metcalfe; Mohan Raizada; Michael J Katovich
Journal:  Curr Hypertens Rep       Date:  2002-02       Impact factor: 5.369

2.  Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia.

Authors:  P T Drapkin; C R O'Riordan; S M Yi; J A Chiorini; J Cardella; J Zabner; M J Welsh
Journal:  J Clin Invest       Date:  2000-03       Impact factor: 14.808

Review 3.  New ideas on the pathophysiology and treatment of lung disease.

Authors:  D F Rogers; G J Laurent
Journal:  Thorax       Date:  1998-03       Impact factor: 9.139

4.  Saturation of, and competition for entry into, the apical secretory pathway.

Authors:  A D Marmorstein; K G Csaky; J Baffi; L Lam; F Rahaal; E Rodriguez-Boulan
Journal:  Proc Natl Acad Sci U S A       Date:  2000-03-28       Impact factor: 11.205

5.  In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors.

Authors:  C Mueller; M S Strayer; J Sirninger; S Braag; F Branco; J-P Louboutin; T R Flotte; D S Strayer
Journal:  Gene Ther       Date:  2009-11-05       Impact factor: 5.250

  5 in total

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