ALS and frontotemporal dementia (FTD) are two neurodegenerative diseases with a toxic relationship, according to a new USC Stem Cell study published in Nature Medicine.
Feb 5, 2018
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127
Respiratory infections peak during the winter months, and most people recover within a few weeks. But for those with a rare genetic lung disease, the sniffling, coughing and congestion never end. The tiny hairlike structures ...
Jan 24, 2018
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40
University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig's disease, in mice, extending their ...
Dec 20, 2017
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A new treatment for Huntington's disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took ...
Dec 14, 2017
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Inside cells, protein "motors" act like trucks on tiny cellular highways to deliver life-sustaining cargoes.
Nov 9, 2017
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A common feature of neurodegenerative diseases is the formation of permanent tangles of insoluble proteins in cells. The beta-amyloid plaques found in people with Alzheimer's disease and the inclusion bodies in motor neurons ...
Jul 27, 2017
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Harvard Stem Cell Institute (HSCI) researchers have identified a compound that helps protect the cells destroyed by spinal muscular atrophy (SMA), the most frequent fatal genetic disease in children under 2 years of age.
Apr 17, 2017
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A new rare muscle disorder has been identified by researchers at Ruhr-Universität Bochum (RUB). This hereditary disease is caused by a defect in the BICD2 gene that manifests itself in altered cellular transport processes ...
Mar 22, 2017
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A puzzling question has lurked behind SMA (spinal muscular atrophy), the leading genetic cause of death in infants.
Feb 14, 2017
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39
Charcot-Marie-Tooth disease is an inherited disorder that leads to a gradual loss of motor neurons and, eventually, paralysis. The condition is caused by genetic mutations that disrupts cells' energy factories, called mitochondria. ...
Oct 24, 2016
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