Progresses towards safe and efficient gene therapy vectors

S Chira, CS Jackson, I Oprea, F Ozturk… - …, 2015 - pmc.ncbi.nlm.nih.gov
The emergence of genetic engineering at the beginning of the 1970′ s opened the era of
biomedical technologies, which aims to improve human health using genetic manipulation …

New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls

SC Bell, K De Boeck, MD Amaral - Pharmacology & therapeutics, 2015 - Elsevier
With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic
defects of cystic fibrosis (CF) commenced. Pharmacological manipulation provides the …

[HTML][HTML] Cystic fibrosis gene therapy: looking back, looking forward

AL Cooney, PB McCray Jr, PL Sinn - Genes, 2018 - mdpi.com
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP …

Gene therapy for cystic fibrosis: Challenges and prospects

H Sui, X Xu, Y Su, Z Gong, M Yao, X Liu… - Frontiers in …, 2022 - frontiersin.org
Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in
a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF …

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

EWFW Alton, JM Beekman, AC Boyd, J Brand… - Thorax, 2017 - thorax.bmj.com
We have recently shown that non-viral gene therapy can stabilise the decline of lung
function in patients with cystic fibrosis (CF). However, the effect was modest, and more …

Advances in gene therapy for cystic fibrosis lung disease

Z Yan, PB McCray Jr… - Human molecular …, 2019 - academic.oup.com
Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts …

Cystic fibrosis gene therapy in the UK and elsewhere

U Griesenbach, KM Pytel, EWFW Alton - Human gene therapy, 2015 - liebertpub.com
The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in
1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which …

Lentiviral-mediated phenotypic correction of cystic fibrosis pigs

AL Cooney, MH Abou Alaiwa, VS Shah… - JCI …, 2016 - pmc.ncbi.nlm.nih.gov
Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in CF
transmembrane conductance regulator (CFTR), resulting in defective anion transport …

Barriers to inhaled gene therapy of obstructive lung diseases: A review

N Kim, GA Duncan, J Hanes, JS Suk - Journal of Controlled Release, 2016 - Elsevier
Abstract Knowledge of genetic origins of obstructive lung diseases has made inhaled gene
therapy an attractive alternative to the current standards of care that are limited to managing …

Gene therapy potential for genetic disorders of surfactant dysfunction

AL Cooney, JA Wambach, PL Sinn… - Frontiers in genome …, 2022 - frontiersin.org
Pulmonary surfactant is critically important to prevent atelectasis by lowering the surface
tension of the alveolar lining liquid. While respiratory distress syndrome (RDS) is common in …