Researchers have developed a promising new approach for gene therapy of inherited blood disorders that may help overcome therapeutically limiting human stem cell gene transfer efficiency. This method would be applicable to patients with beta-thalassemia, a potentially life-threatening blood disease, as well as other genetic blood disorders, according to a study published in the July 15th issue of Blood. By transplanting beta-thalassemic mice with stem cells treated with MGMT (methylguanine methyltransferase), a drug-resistance gene, researchers were able to subsequently give a chemotherapy drug to the mice, which specifically increased the normal, or globin-expressing cells, to levels that diminished, or in some cases, cured the disease. The transplanted donor stem cells genetically reversed the beta-thalassemia in the mice because the drug-resistant cells assumed production of normal red blood cells in the bone marrow.
"Our finding gives us hope that we might one day be able to help patients with hemoglobin diseases generate healthy blood cells in their own bodies," said Derek Persons, MD, assistant member in the St. Jude Department of Hematology/Oncology, Memphis, TN, and lead author of the paper. "The technique we pioneered will allow us to enrich the population of cells carrying the normal gene by eliminating competing, defective cells, without using radiation or intensive chemotherapy."
Researchers in the study used an oncoretrovirus to transfer MGMT into normal bone marrow cells. The treated cells were then transplanted into beta-thalassemic mice previously given non-myeloablative (non-life-threatening) pre-transplant conditioning with the cytotoxic agents temozolomide (TMZ) and O6-benzylguanine (BG). The transplanted mice were randomly assigned to receive either drug treatment with TMZ/BG (two five-day courses, five weeks apart, beginning seven weeks after transplant) or no treatment.
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Friction stir welding is a still-young and thus often unfamiliar pressure welding process for joining flat components and semi-finished components made of light metals.
Scientists at the University of Stuttgart have now developed two new process variants that will considerably expand the areas of application for friction stir welding.
Technologie-Lizenz-Büro (TLB) GmbH supports the University of Stuttgart in patenting and marketing its innovations.
Friction stir welding is a still-young and thus often unfamiliar pressure welding process for joining flat components and semi-finished components made of...
Optical quantum computers can revolutionize computer technology. A team of researchers led by scientists from Münster University and KIT now succeeded in putting a quantum optical experimental set-up onto a chip. In doing so, they have met one of the requirements for making it possible to use photonic circuits for optical quantum computers.
Optical quantum computers are what people are pinning their hopes on for tomorrow’s computer technology – whether for tap-proof data encryption, ultrafast...
The Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP has been developing various applications for OLED microdisplays based on organic semiconductors. By integrating the capabilities of an image sensor directly into the microdisplay, eye movements can be recorded by the smart glasses and utilized for guidance and control functions, as one example. The new design will be debuted at Augmented World Expo Europe (AWE) in Berlin at Booth B25, October 18th – 19th.
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With the help of artificial intelligence, chemists from the University of Basel in Switzerland have computed the characteristics of about two million crystals made up of four chemical elements. The researchers were able to identify 90 previously unknown thermodynamically stable crystals that can be regarded as new materials. They report on their findings in the scientific journal Physical Review Letters.
Elpasolite is a glassy, transparent, shiny and soft mineral with a cubic crystal structure. First discovered in El Paso County (Colorado, USA), it can also be...
For the first time, Fraunhofer IKTS shows additively manufactured hardmetal tools at WorldPM 2016 in Hamburg. Mechanical, chemical as well as a high heat resistance and extreme hardness are required from tools that are used in mechanical and automotive engineering or in plastics and building materials industry. Researchers at the Fraunhofer Institute for Ceramic Technologies and Systems IKTS in Dresden managed the production of complex hardmetal tools via 3D printing in a quality that are in no way inferior to conventionally produced high-performance tools.
Fraunhofer IKTS counts decades of proven expertise in the development of hardmetals. To date, reliable cutting, drilling, pressing and stamping tools made of...
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29.09.2016 | Materials Sciences
29.09.2016 | Materials Sciences
29.09.2016 | Interdisciplinary Research