St. Jude Children's Research Hospital scientists have discovered how an aggressive tumor of the muscle and soft tissue hijacks an export pathway in cells to lay the groundwork for cancer progression and resistance to chemotherapy. ...
Dec 21, 2015
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64
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. The key may be to inject viruses ...
Nov 11, 2015
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427
Two proteins that share the ability to help cells deal with their trash appear to need each other to do their jobs and when they don't connect, it appears to contribute to development of Parkinson's disease, scientists report.
Aug 20, 2015
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167
Lysosomes, the "garbage disposal" systems of cells, are found in great abundance near the amyloid plaques in the brain that are a hallmark of Alzheimer's disease. Scientists have long assumed that their presence was helpful—that ...
Jun 30, 2015
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A first-in-man clinical trial, testing a newly developed drug for a rare genetic condition called mucopolysaccharidosis type II (MPS II) or Hunter syndrome, is underway at Emory University.
Jun 3, 2015
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36
Researchers have identified a bacterial protein that triggers a self-inflicted cell death pathway in immune system cells and could lead to a better understanding of an important cellular structure.
Mar 17, 2015
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67
Scientists at The Scripps Research Institute (TSRI) have identified a new cellular pathway that is affected in cystinosis, a rare genetic disorder that can result in eye and kidney damage.
Feb 10, 2015
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9
Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal ...
Jan 7, 2015
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6
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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A European Journal of Neuroscience study suggests that it is critical to treat lysosomal storage disorders early, before symptoms arise. These genetic disorders, which are caused by the malfunction of enzymes that normally ...
Aug 4, 2014
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