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Keywords: gene therapy
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Biochem Soc Trans (2021) 49 (6): 2539–2548.
Published: 09 December 2021
... 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...
Includes: Supplementary data
Biochem Soc Trans (2021) 49 (5): 2443–2454.
Published: 28 October 2021
... and with the advancements of gene therapies for rare diseases in recent years [ 16 , 83 , 84 ], LUBAC mediated PB disorders could benefit from gene replacement approaches. The observation that the polyglucosans phenotype in patients and in mouse models have been primarily associated with mutations in HOIL-1L...
Biochem Soc Trans (2018) 46 (5): 1247–1261.
Published: 09 October 2018
... 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...
Biochem Soc Trans (2018) 46 (1): 11–21.
Published: 01 December 2017
... on behalf of the Biochemical Society 2018 cardiovascular disease gene therapy high-throughput screening microRNA The foundation of molecular biology states that DNA is copied to RNA by the process of transcription. The transcribed RNA then translates the information to produce proteins...
Biochem Soc Trans (2016) 44 (5): 1483–1490.
Published: 19 October 2016
... Attribution License 4.0 (CC BY) . gene therapy mitochondria mitochondrial dysfunction mouse models therapeutics The main function of mitochondria is to convert the energy derived from nutrients into heat and ATP, a high-energy molecule exploited by the cell biochemical machineries...
Biochem Soc Trans (2016) 44 (5): 1355–1365.
Published: 19 October 2016
... 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...
Biochem Soc Trans (2011) 39 (2): 584–588.
Published: 22 March 2011
... cleavages. 1 To whom correspondence should be addressed (email s.halford@bristol.ac.uk ). DNA looping gene therapy protein–protein interaction recognition sequence restriction enzyme Type II restriction endonucleases recognize specific DNA sequences, typically 4–8 bp long...
Biochem Soc Trans (2010) 38 (6): 1484–1488.
Published: 24 November 2010
...-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...
Biochem Soc Trans (2010) 38 (6): 1489–1493.
Published: 24 November 2010
... ). 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 AAV adeno-associated virus CNS central nervous system dpc days post-coitus EIAV...
Biochem Soc Trans (2009) 37 (6): 1198–1200.
Published: 19 November 2009
... to a recent review covering these areas [ 3 ]. 1 email seppo.ylaherttuala@uku.fi 7 8 2009 © The Authors Journal compilation © 2009 Biochemical Society 2009 angiogenesis arteriogenesis gene therapy ischaemia lymphangiogenesis vascular endothelial growth factor (VEGF...
Biochem Soc Trans (2003) 31 (2): 397–406.
Published: 01 April 2003
... 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...