... the cardiac damage associated with diabetes. One potential alternative avenue for targeting diabetes-induced heart failure is the use of adeno-associated viral vector (AAV) gene therapy, which has shown great versatility in a multitude of disease settings. AAV gene therapy has the potential to target specific...

... apoptosis. However, by overexpressing CD59, a membrane complement regulatory protein (mCRP), lung cancer cells develop resistance to CDC. We previously showed that virus-like particles (VLPs) of human JC polyomavirus (JCPyV) could be used as a gene therapy vector to carry a suicide gene expression plasmid...

.... The specific role of MCAD in the adult heart is unknown. To examine the role of MCAD in the heart and to assess the therapeutic potential of increasing MCAD in the failing heart, we developed a gene therapy tool using recombinant adeno-associated viral vectors (rAAV) encoding MCAD. We hypothesised...

... of the Biochemical Society 2017 gene therapy cystic fibrosis transmembrane conductance regulator exercise Cystic fibrosis (CF) results from mutation of the gene which encodes Cystic Fibrosis Transmembrane conductance Regulator protein (CFTR), a chloride ion channel [ 1 ]. The G551D mutation (Class...

... 04 2017 © 2017 The Author(s). Published by Portland Press Limited on behalf of the Biochemical Society 2017 epilepsy excitable membranes gene therapy neurological disorders optogenetics translational science Epilepsies are a heterogeneous group of diseases, characterized...

... cardiac oxidative stress and dysfunction. In the present study, we investigated the therapeutic potential of a delayed intervention with cardiac-targeted PI3K gene therapy, administered to mice with established diabetes-induced LV diastolic dysfunction. Diabetes was induced in 6-week-old male mice...

... Amalia Forte (email [email protected] ). 21 2 2014 22 4 2014 19 5 2014 © The Authors Journal compilation © 2014 Biochemical Society 2014 animal model gene therapy microRNA stem cell pharmacogenomics restenosis Cardiovascular diseases include myocardial...

... was compared with that of constitutively expressed β-actin. adenovirus gene therapy haem oxygenase ischaemia/reperfusion ocular disease retinal ganglion cell Retinal IR (ischaemia/reperfusion) injury, leading to RGC (retinal ganglion cell) death, is a common aetiology accounting...

... the progress made and the pitfalls encountered to date, and critically assesses the evidence for the use of stem cells in lung disease. airway bone marrow gene therapy lung disease epithelial cell progenitor stem cell Although the endogenous repair and continual tissue replacement by local...

... 28 6 2005 The Biochemical Society 2006 clinical trial gene therapy Huntington's disease neurodegeneration neurotransplantation stem cell treatment HD (Huntington's disease) is an inherited neurodegenerative disorder, with the most common age of onset between 30–50 years...

... replacement therapy have not fulfilled earlier promises, although there is still interest in other dietary supplements. Superoxide dismutase mimetics, thiols, xanthine oxidase and NAD(P)H oxidase inhibitors are currently receiving much interest, while animal studies using gene therapy show promise...

... gene expression in cells of the outer retina. Recent progress has enabled the planning of clinical trials of gene therapy for ocular neovascular disorders. angiogenesis gene therapy neovascularization recombinant adeno-associated virus retina * This paper was presented...

...Robin H. LACHMANN; Stacey EFSTATHIOU Gene therapy might provide a useful treatment for a number of neurological diseases and a great deal of effort is going into the development of vector systems which will allow the delivery of potentially therapeutic genes to terminally differentiated neurons...