Drugs that manipulate the code of life are finally changing lives Source Scientific American The idea for gene therapy—a type of DNA-based medicine that inserts a healthy gene into cells to replace a mutated, disease-causing variant—was first published in 1972. After decades of disputed results, treatment failures and some deaths in experimental trials, the first
The strategy has long been seen as having enormous potential to cure diseases Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. The feat, published in the journal Nature Biotechnology, offers
Researchers from EMBL and the German Cancer Research Center (DKFZ) in Heidelberg have developed new methods to reveal the three-dimensional organisation of bone marrow at a single cell level. Since bone marrow harbours blood stem cells responsible for life-long blood generation, these results and the new method provides a novel scientific basis to study blood
Mutations in the RIPK1 gene responsible for CRIA syndrome. Over the last 20 years, three families have been unsuspectingly linked by an unknown illness. Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and other organizations have now identified the cause of the illness, a new disease called
Potential 1st Gene Therapy in Europe Directed at Any Type of Hemophilia Application to be Reviewed Under Accelerated Assessment BioMarin Pharmaceutical announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January
A research team at Nanyang Technological University, Singapore (NTU Singapore) has found an easier way to harvest healing factors – molecules that promote tissue growth and regeneration – from adult stem cells Presently, scientists ‘pre-condition’ adult stem cells to secrete healing factors by putting them in a low-oxygen chamber or by using biochemicals or genetic engineering.However in
After decades of research – much of it funded by the public – gene therapy is a reality. Now high costs are blocking patient access. Prices should be constrained Source Science Business Gene therapy is providing unprecedented hope for growing numbers of patients and families. This game changer in medicine restores vision in babies born
GE Healthcare Life Sciences and Advanced Solutions Life Sciences (ASLS), a Kentucky-based biotechnology firm, are collaborating to build an integrated 3D bioprinter by combining their respective technologies. ASLS’ BioAssemblyBot 3D bioprinter will be integrated with GE Healthcare Life Sciences’ IN Cell Analyzer confocal imaging platform, as part of a strategic R&D and distribution partnership. By combining the two technologies, the
The ISLET project is a multi-center collaboration of leading European researchers that has been awarded 8 million Euros in a 5-year period. The project will develop advanced stem cell products ready for use in clinical trials in Type 1 Diabetes. Professor Henrik Semb from DanStem is coordinating the project. The ISLET project will develop a
Scientists at the University of St Andrews and the University of Edinburgh have identified a specific type of cell in the human body which can cause motor neurons to fail using stem cell technology. The joint research, published today (Monday 16 December) in Glia, has shown that glial cells, which normally support neurons throughout the brain and spinal
