... of the Biochemical Society and distributed under the Creative Commons Attribution License 4.0 (CC BY) . alpha cell diabetes gene therapy Type 1 diabetes is a chronic disease characterized by autoimmune-mediated destruction of the insulin-producing β cells in the pancreas [ 1 ]. Destruction of β...

... for this article was enabled by the participation of University of Texas Southwestern Medical Center in an all-inclusive Read & Publish pilot with Portland Press and the Biochemical Society under a transformative agreement with EBSCO. gene therapy HOIL-1L HOIP LUBAC M1 ubiquitination ubiquitin...

... syndrome; Red arrow, gene therapy; Green arrow, drug approval process; Blue arrow, drug compound; *= GMP product development. mTORC1 is a cytosolic Ser/Thr kinase belonging to the phosphatidylinositol kinase-related protein kinases family with central roles in several cellular processes...

... screening. We also discuss the challenge to achieve a safe and targeted delivery of miRNA therapeutics in cardiovascular cells. cardiovascular disease gene therapy high-throughput screening microRNA This diverse range of biological effects has made them a tempting target for drug discovery...

... Limited on behalf of the Biochemical Society and distributed under the Creative Commons Attribution License 4.0 (CC BY) . gene therapy mitochondria mitochondrial dysfunction mouse models therapeutics Beside mtDNA-encoded proteins, the vast majority of the ∼1500 polypeptides forming...

... and adverse effects. Therefore, more durable therapeutic strategies should be considered, such as a stable gene therapy to protect the target T cells against HIV-1 infection. The development of potent therapeutic regimens based on the RNA interference (RNAi) and clustered regularly interspaced short...

... Present address: Division of Molecular Microbiology, College of Life Sciences, University of Dundee, Dundee DD1 5EH, U.K. Pairs of ZFNs designed to act at two adjacent sequences have been widely advocated as potential tools for targeting individual genes and for gene therapy [ 43 – 45...

... ). 15 6 2010 © The Authors Journal compilation © 2010 Biochemical Society 2010 gene therapy in utero gene transfer lysosomal storage disorder mouse nervous system viral vector Early lethal genetic diseases of the nervous system are individually rare, yet collectively numerous...

...-like growth factor) II receptor provides an avenue for endocytosis, trafficking and lysosomal processing of extracellularly delivered enzyme. The present review discusses therapeutic utilization of cross-correction by enzyme-replacement therapy, gene therapy and stem cell therapy for the NCLs, along...

... angiogenesis arteriogenesis gene therapy ischaemia lymphangiogenesis vascular endothelial growth factor (VEGF) References 1 Ylä-Herttuala S. Alitalo K. Gene transfer as a tool to induce therapeutic vascular growth Nat. Med. 2003 9 694 701 2 Alitalo K. Tammela T...

... molecular probes as tools to throw light on the problematic steps in non-viral gene delivery which still impede efficient gene therapy. Thus, the current aims of our research are to understand, design and prepare small-molecule lipopolyamines for non-viral gene therapy (NVGT). The rational design...