The discovery of a gene mutation that causes a rare premature aging disease could lead to the development of drugs that block the rapid, unstoppable cell division that makes cancer so deadly.
Sep 30, 2014
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REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
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Researchers have discovered previously undetected steps in the process by which stem cells become blood cells, a process called haematopoiesis. An international collaboration, including Winthrop Professor Wendy Erber, has ...
Sep 26, 2014
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A study published on September 25th in PLOS Pathogens reports a new primate model to test treatments that might cure HIV/AIDS and suggests answers to questions raised by the "Berlin patient", the only human thought to have ...
Sep 25, 2014
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Investigators from the Institute for Research in Immunology and Cancer (IRIC) at the Université de Montréal have just published, in the prestigious magazine Science, the announcement of the discovery of a new molecule, ...
Sep 18, 2014
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Two female patients achieved clinical remission from the rare, debilitating neurological disease called stiff person syndrome (SPS, which can be marked by a "tin soldier" gait) after an autologous (from your own body) stem ...
Aug 25, 2014
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Hematopoietic stem cells (HSCs) give rise to all other blood cell types, but their development and how their fate is determined has long remained a mystery. In a paper published online this week in Nature, researchers at ...
Aug 14, 2014
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A major hurdle in gene therapy is the efficient integration of a corrected gene into a patient's genome without mutating off-target sites. In a paper published today in Genome Research, scientists have used CRISPR/Cas genome ...
Aug 5, 2014
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There's a good reason people over 60 are not donor candidates for bone marrow transplantation. The immune system ages and weakens with time, making the elderly prone to life-threatening infection and other maladies, and a ...
Jul 31, 2014
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The ability to reliably and safely make in the laboratory all of the different types of cells in human blood is one key step closer to reality.
Jul 14, 2014
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