Gene Therapy

News Gene Therapy

• European Medicines Agency publishes 2012 annual report

  The European Medicines Agency has published its 2012 annual report. The report highlights the main trends recently observed in the Agency’s activities. For the past few years, the Agency has received a stable number of initial marketing authorisation applications (MAAs) for human medicines, with a t more

• First acute intermittent porphyria patient treated with a gene therapy product

  Digna Biotech sets the first clinical trial in Spain to treat acute intermittent porphyria, a rare genetic disease that affects the biosynthesis of heme (a component of hemoglobin) and can cause significant neurological damage. This biotechnology company coordinates a phase I clinical trial in colla more

• Danish chemist aims to bring supermolecules to the world

  With applications spanning from non-shrink dental fillings to DNA-drugs the so-called dendrimers are a near magical material. Now a chemist from the University of Copenhagen has vowed to make the weird molecules famous. When a molecule's structure resembles that of two tree crowns growing into one a more

White papers Gene Therapy

• Optimized Production of Lentivirus Using FuGENE® HD Transfection Reagent
  

  Lentiviral vectors are ideally suited vehicles for a broad range of research applications. Depending on pseudotype, lentiviruses infect a wide range of cell types, non-discriminately transducing both dividing and non-dividing cells. As opposed to other popular vector delivery systems, lentivirus stably and rapidly integrates genetic payload into the host genome allowing for long-term studies in vivo. Current lentiviral vector systems can accommodate upwards of ten kilobases of foreign DNA [1], although promoter and enhancer elements reduce the practical size of gene open-reading frames to perhaps five or six kilo­bases, which is sufficient to accommodate most genes commonly studied in the mammalian genome. more

• Advances in Transfection Technologies: Using FuGENE® HD Transfection Reagent with PCR Constructs
  

  With recent advances in molecular biology, it is now possible to construct genomic sequences and then evaluate the protein produced by those sequences. Ever-increasing portions of the genome are being investigated for their functions or for the effect sequence changes might have on them. Such study may entail testing the function of an unknown region of the genome, or mutating a sequence in a particular region to see how protein function or binding to other molecules might be affected. In these cases, using a complete plasmid for transfection is tedious, requiring time-consuming cloning, amplification, and purification of a plasmid construct. If multiple variants are to be tested, it would be much simpler if the PCR product could be tested directly, and a plasmid construct made only once the sequence of interest is identified. more

• FuGENE® HD Transfection Reagent Provides High Transfection Efficiency in the Murine NB2a Cell Line
  

  Cell transfection is a method of gene delivery that introduces foreign DNA into a cell. It is one of the most important approaches in modern genetic and proteomic research. With intriguing properties, transfection of primary cells is required for tissue engineering and gene therapy. However, one major problem is that many ­methods do not work for neural cell lines. Here we report that FuGENE® HD Transfection Reagent provides a high level of transfection efficiency and intracellular protein expression in murine neuroblastoma cells, the NB2a cell line. An optimization test using different transfection reagents, including FuGENE® HD Transfection Reagent, FuGENE® 6 Transfection Reagent, and another liposome-mediated reagent, was performed for green fluores­cent protein (GFP) expression in NB2a cells through transient transfection. more

Publications Gene Therapy

• Viruses, Vol. 5, Pages 1500-1515: The Role of Chromatin in Adenoviral Vector Function

  Vectors based on adenovirus (Ad) are one of the most commonly utilized platforms for gene delivery to cells in molecular biology studies and in gene therapy applications. Ad is also the most popular vector system in human clinical gene therapy trials, largely due to its advantageous characteristics more

• Beneficial effects of selective HDL-raising gene transfer on survival, cardiac remodelling and cardiac function after myocardial infarction in mice

  Beneficial effects of selective HDL-raising gene transfer on survival, cardiac remodelling and cardiac function after myocardial infarction in mice Gene Therapy advance online publication, June 13 2013. doi:10.1038/gt.2013.30 Authors: S C Gordts, I Muthuramu, E Nefyodova, F Jacobs, E more

• Role of antigen-specific regulatory CD4|[plus]|CD25|[plus]| T cells in tolerance induction after neonatal IP administration of AAV-hF.IX

  Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX Gene Therapy advance online publication, June 13 2013. doi:10.1038/gt.2013.22 Authors: Y Shi, R Falahati, J Zhang, L Flebbe-Rehwaldt & K M L more

Market studies Gene Therapy

• Translational Regenerative Medicine - Oncology, CNS and Cardiovascular-Rich Pipeline Features Innovative Stem Cell and Gene Therapy Applications

  GBI Research, the leading business intelligence provider, has released its latest research, “Translational Regenerative Medicine - Oncology, CNS and Cardiovascular-Rich Pipeline Features Innovative Stem Cell and Gene Therapy Applications”, which provides insights into the global regenerative medicin more

• Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

  GBI Research’s new report, “Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards”, provides in-depth analysis of the unmet needs, drivers and barriers that impact the global gene therapy market. The report analyzes more

• Global Hemophilia Market Report: 2012 Edition

  With an ever changing technological environment and an unstable economic scenario, many scientific studies are being conducted which are leading to the discovery of new coagulation factors, proteins and plasma concentrates used for treating chronic diseases and medical conditions. The market for hem more

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