In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short ...
Nov 21, 2022
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The innate immune system fights infectious agents, and the alarm messenger interferon plays a central role in this. However, if interferon is produced in the absence of an infection, it can lead to autoimmune diseases. Researchers ...
Nov 8, 2022
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Scientists have shown how stomach cancers can dodge the effects of a promising new experimental treatment—raising the possibility of outsmarting cancer by predicting drug resistance in advance.
Oct 25, 2022
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An Undiagnosed Diseases Network (UDN) study led by Dr. Hugo Bellen, investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital and distinguished service professor at the Baylor ...
Oct 4, 2022
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Primary immunodeficiency disorders (PID) can result in chronic and sometimes life-threatening infections. More than 450 PIDs have been described, but timely and accurate diagnoses remain a challenge. In a new study in The ...
May 23, 2022
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A newly discovered gene defect among people of Inuit ancestry in Greenland, Canada and Alaska will possibly lead to screening of all newborn Inuits as they will otherwise be at risk of dying from child vaccines or simple ...
Apr 26, 2022
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Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease. ...
Jan 4, 2022
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Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
Aug 3, 2021
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Cancer and many other diseases are based on genetic defects. The body can often compensate for the defect of one gene; it is only the combination of several genetic errors that leads to the clinical picture. The 3Cs multiplex ...
Jun 24, 2021
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UNC School of Medicine scientists led a collaboration of researchers to demonstrate a potentially powerful new strategy for treating cystic fibrosis (CF) and potentially a wide range of other diseases. It involves small, ...
Jun 16, 2021
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