Genetic modification of the lung directed toward treatment of human disease

D Sondhi, KM Stiles, BP De, RG Crystal - Human gene therapy, 2017 - liebertpub.com
Genetic modification therapy is a promising therapeutic strategy for many diseases of the
lung intractable to other treatments. Lung gene therapy has been the subject of numerous …

Gene therapy for alpha-1 antitrypsin deficiency lung disease

MJ Chiuchiolo, RG Crystal - Annals of the American Thoracic …, 2016 - atsjournals.org
Alpha-1 antitrypsin (AAT) deficiency, characterized by low plasma levels of the serine
protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of …

5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency

C Mueller, G Gernoux, AM Gruntman, F Borel… - Molecular Therapy, 2017 - cell.com
Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due
principally to the unopposed effects of neutrophil elastase. We previously reported achieving …

Biosurfactants of MEL-A increase gene transfection mediated by cationic liposomes

Y Inoh, D Kitamoto, N Hirashima… - … and Biophysical Research …, 2001 - Elsevier
Many microorganisms growing on water-insoluble substrates have been known to produce
surface-active compounds called biosurfactants. Although biosurfactants have received …

Long-term therapeutic levels of human alpha-1 antitrypsin in plasma after hydrodynamic injection of nonviral DNA

SF Aliño, A Crespo, F Dasi - Gene Therapy, 2003 - nature.com
The transfection efficacy of several vectors containing the full genomic hAAT gene with its
natural promoter (pTG7101) and others containing the cDNA of hAAT gene driven by …

Cationic lipid-mediated gene transfer: analysis of cellular uptake and nuclear import of plasmid DNA

V Escriou, C Ciolina, A Helbling-Leclerc, P Wils… - Cell biology and …, 1998 - Springer
Cationic lipids are widely used for gene transfer in vitro and show promise as vectors for in
vivo gene therapy applications. However, there is limited understanding of the cellular …

Translational advances of hydrofection by hydrodynamic injection

L Sendra, MJ Herrero, SF Aliño - Genes, 2018 - mdpi.com
Hydrodynamic gene delivery has proven to be a safe and efficient procedure for gene
transfer, able to mediate, in murine model, therapeutic levels of proteins encoded by the …

Anionic liposomes increase the efficiency of adenovirus-mediated gene transfer to coxsackie-adenovirus receptor deficient cells

Z Zhong, S Shi, J Han, Z Zhang, X Sun - Molecular pharmaceutics, 2010 - ACS Publications
Despite remarkable progress in the research of both viral and nonviral gene delivery
vectors, the drawbacks in each delivery system have limited their clinical applications …

Gene therapy for liver diseases: recent strategies for treatment of viral hepatitis and liver malignancies

V Schmitz, C Qian, J Ruiz, B Sangro, I Melero… - Gut, 2002 - gut.bmj.com
Gene therapy has emerged as a powerful and very plastic tool to regulate biological
functions in diseased tissues with application in virtually all medical fields. An increasing …

Asialofetuin liposome-mediated human α1-antitrypsin gene transfer in vivo results in stationary long-term gene expression

F Dasi, M Benet, J Crespo, A Crespo… - Journal of molecular …, 2001 - Springer
The development of nonviral vectors for in vivo gene delivery to hepatocytes is an interesting
topic in view of their safety and tremendous gene therapy potential. Since cationic liposomes …