In pursuit of new developments for gene therapy of human diseases

G Palu, R Bonaguro, A Marcello - Journal of biotechnology, 1999 - Elsevier
Gene therapy aims at transferring a therapeutic gene into human somatic cells in order to
treat a disease. Originally addressed to hereditary genetic disorders, gene therapy has …

[HTML][HTML] Delivery systems for gene therapy

S Mali - Indian journal of human genetics, 2013 - ncbi.nlm.nih.gov
The structure of DNA was unraveled by Watson and Crick in 1953, and two decades later
Arber, Nathans and Smith discovered DNA restriction enzymes, which led to the rapid …

[引用][C] Progress toward human gene therapy

AD Miller - 1990 - ashpublications.org
The material transferred is on the order of one to a few genes in size. Since many hereditary
diseases are caused by defects in single genes, there are many potential applications of this …

[HTML][HTML] Gene therapy, early promises, subsequent problems, and recent breakthroughs

SR Soofiyani, B Baradaran, F Lotfipour… - Advanced …, 2013 - ncbi.nlm.nih.gov
Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to
tissues for clinical applications has been discussed around half a century, but scientist's …

The basic science of gene therapy

RC Mulligan - Science, 1993 - science.org
The development over the past decade of methods for delivering genes to mammalian cells
has stimulated great interest in the possibility of treating human disease by gene-based …

Prospects and problems of gene therapy: an update

DC Gorecki - Expert opinion on emerging drugs, 2001 - Taylor & Francis
The gene therapy approach can vary from delivering extra copies of a gene, through
modifications of a genome using the properties of ribozymes or chimeraplasts, to injection of …

Gene therapy: the first decade

A Mountain - Trends in biotechnology, 2000 - cell.com
Gene therapy promises to revolutionize medicine by treating the causes of disease rather
than the symptoms. We are nearing the end of the first decade of gene therapy, and this …

[HTML][HTML] Conceptual and technical aspects of transfection and gene delivery

L Kaestner, A Scholz, P Lipp - Bioorganic & medicinal chemistry letters, 2015 - Elsevier
Genetically modified animals are state of the art in biomedical research as gene therapy is a
promising perspective in the attempt to cure hereditary diseases. Both approaches have in …

[HTML][HTML] Human gene therapy: novel approaches to improve the current gene delivery systems

M Cucchiarini - Discovery Medicine, 2016 - discoverymedicine.com
Even though gene therapy made its way through the clinics to treat a number of human
pathologies since the early years of experimental research and despite the recent approval …

Somatic gene therapy. Present situation and future perspective.

U Gottschalk, S Chan - Arzneimittel-forschung, 1998 - europepmc.org
The ultimate goal in the management of inherited as well as acquired diseases is a rational
therapy with the aim to eliminate the underlying biochemical defects, rather than a …