(Medical Xpress) -- Scientists have used a gene therapy tool that acts like intelligent molecular scissors to correct the key gene defect in mice with hemophilia B, a disease that can lead to uncontrolled bleeding. The intervention ...
Jun 28, 2011
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Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young adults have been fixed in patient-derived kidney cells using a potentially game-changing DNA repair-kit. The advance, developed ...
Jul 29, 2022
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The oral drug ivacaftor appears to be safe and could be beneficial to young children between the ages of 2 and 5 with a specific type of cystic fibrosis, according to new research published in The Lancet Respiratory Medicine ...
Jan 21, 2016
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A new study from BC Children's Hospital, the University of British Columbia (UBC) and an international team of researchers published in the New England Journal of Medicine is the first to identify a rarely-seen type of DNA ...
Apr 15, 2019
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As the medical community continues to make positive strides in personalized cancer therapy, scientists know some dead ends are unavoidable. Drugs that target specific genes in cancerous cells are effective, but not all proteins ...
Aug 7, 2012
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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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Huntington's disease is a fatal condition involving the death of brain cells, typically striking in midlife. But new findings suggest the disease process starts decades earlier. Although symptoms emerge in adulthood, researchers ...
Oct 5, 2021
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Researchers studying rare genetic disorders have uncovered insights into those diseases in biological structures that regulate chromosomes when cells divide. Focusing on the cohesin complex, a group of proteins forming a ...
May 29, 2012
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A single genetic defect on the X chromosome that can result in a wide array of conditions—from learning and emotional difficulties to primary ovarian insufficiency in women and tremors in middle-aged men—occurs at a much ...
Dec 20, 2012
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A new study from an international research team, led by Dr. Yalda Jamshidi at St George's, University of London, has identified new genes associated with heart function and development.
Jul 17, 2018
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