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.
How cancer metastasis happens: Researchers reveal a key mechanism
19.01.2018 | Weill Cornell Medicine
Researchers identify new way to unmask melanoma cells to the immune system
17.01.2018 | Duke University Medical Center
On the way to an intelligent laboratory, physicists from Innsbruck and Vienna present an artificial agent that autonomously designs quantum experiments. In initial experiments, the system has independently (re)discovered experimental techniques that are nowadays standard in modern quantum optical laboratories. This shows how machines could play a more creative role in research in the future.
We carry smartphones in our pockets, the streets are dotted with semi-autonomous cars, but in the research laboratory experiments are still being designed by...
What enables electrons to be transferred swiftly, for example during photosynthesis? An interdisciplinary team of researchers has worked out the details of how...
For the first time, scientists have precisely measured the effective electrical charge of a single molecule in solution. This fundamental insight of an SNSF Professor could also pave the way for future medical diagnostics.
Electrical charge is one of the key properties that allows molecules to interact. Life itself depends on this phenomenon: many biological processes involve...
At the JEC World Composite Show in Paris in March 2018, the Fraunhofer Institute for Laser Technology ILT will be focusing on the latest trends and innovations in laser machining of composites. Among other things, researchers at the booth shared with the Aachen Center for Integrative Lightweight Production (AZL) will demonstrate how lasers can be used for joining, structuring, cutting and drilling composite materials.
No other industry has attracted as much public attention to composite materials as the automotive industry, which along with the aerospace industry is a driver...
Scientists at Tokyo Institute of Technology (Tokyo Tech) and Tohoku University have developed high-quality GFO epitaxial films and systematically investigated their ferroelectric and ferromagnetic properties. They also demonstrated the room-temperature magnetocapacitance effects of these GFO thin films.
Multiferroic materials show magnetically driven ferroelectricity. They are attracting increasing attention because of their fascinating properties such as...
08.01.2018 | Event News
11.12.2017 | Event News
08.12.2017 | Event News
22.01.2018 | Materials Sciences
22.01.2018 | Earth Sciences
22.01.2018 | Life Sciences