scholarly journals Integrating Viral and Nonviral Vectors for Cystic Fibrosis Gene Therapy in the Airways

10.5772/60977 ◽  
2015 ◽  
Author(s):  
Ashley L. Cooney ◽  
Paul B. McCray ◽  
Patrick L. Sinn
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
Nonviral Vectors

Use of Nonviral Vectors for Cystic Fibrosis Gene Therapy

2004 ◽  
Vol 1 (4) ◽  
pp. 296-301 ◽  
Author(s):  
E. W. F. W. Alton
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
Nonviral Vectors

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

2014 ◽  
pp. 150127063140004
Author(s):  
Lee Adrian Davies ◽  
Graciela A Nunez-Alonso ◽  
Gerry McLachlan ◽  
Stephen C Hyde ◽  
Deborah Rebecca Gill
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
Aerosol Delivery

Cystic fibrosis gene therapy

1994 ◽  
Vol 4 (3) ◽  
pp. 466-471 ◽  
Author(s):  
William H. Colledge
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene

scholarly journals Cystic Fibrosis Gene Therapy Trials and Tribulations

2000 ◽  
Vol 1 (1) ◽  
pp. 5-6 ◽  
Author(s):  
Pamela L. Zeitlin
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene

Biology students' understanding of cystic fibrosis, gene therapy, and gene screening

1998 ◽  
Vol 32 (2) ◽  
pp. 103-110 ◽  
Author(s):  
Ruaraidh Hill ◽  
Helen O'Sullivan ◽  
Martin Stanisstreet ◽  
Edward Boyes
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
Biology Students ◽  
Gene Screening

Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy

2018 ◽  
Vol 29 (6) ◽  
pp. 641-642 ◽  
Author(s):  
Terence R. Flotte
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
Basal Cells

Cystic Fibrosis: Gene Therapy Approaches

2003 ◽  
pp. 207-226 ◽  
Author(s):  
Natasha J. Caplen
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene

Cystic fibrosis gene therapy: successes, failures and hopes for the future

2009 ◽  
Vol 3 (4) ◽  
pp. 363-371 ◽  
Author(s):  
Uta Griesenbach ◽  
Eric WFW Alton
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Cystic Fibrosis Gene ◽  
The Future

scholarly journals Cystic Fibrosis Gene Therapy: Looking Back, Looking Forward

Genes ◽  
2018 ◽  
Vol 9 (11) ◽  
pp. 538 ◽  
Author(s):  
Ashley Cooney ◽  
Paul McCray ◽  
Patrick Sinn
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Anion Channel ◽  
Gene Replacement ◽  
Cystic Fibrosis Gene ◽  
Cftr Gene ◽  
System Disease ◽  
Cftr Mutations ◽  
Infection And Inflammation

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel. Although CF is a multi-organ system disease, most people with CF die of progressive lung disease that begins early in childhood and is characterized by chronic bacterial infection and inflammation. Nearly 90% of people with CF have at least one copy of the ΔF508 mutation, but there are hundreds of CFTR mutations that result in a range of disease severities. A CFTR gene replacement approach would be efficacious regardless of the disease-causing mutation. After the discovery of the CFTR gene in 1989, the in vitro proof-of-concept for gene therapy for CF was quickly established in 1990. In 1993, the first of many gene therapy clinical trials attempted to rescue the CF defect in airway epithelia. Despite the initial enthusiasm, there is still no FDA-approved gene therapy for CF. Here we discuss the history of CF gene therapy, from the discovery of the CFTR gene to current state-of-the-art gene delivery vector designs. While implementation of CF gene therapy has proven more challenging than initially envisioned; thanks to continued innovation, it may yet become a reality.

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