Hematopoietic stem cells (HSCs) are rare cells found in the bone marrow that produce red blood cells, white blood cells, and platelets. Their correct functioning is indispensable for the growth and health of an organism. ...
Jul 10, 2023
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Tafamidis meglumine (trade name: Vyndaqel) was approved in November 2011 for the treatment of transthyretin amyloidosis in adults. This rare disorder ("orphan disease") is caused by a defective gene and is associated with ...
Sep 17, 2012
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To mend a broken heart—that is, to regenerate a damaged cardiac muscle—it helps to know how hearts are built. "How does one stem cell, which has no specific identity, develop into multiple cell types that organize into ...
Aug 4, 2015
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Uterine leiomyomata, or fibroids, are benign tumours that nevertheless affect the health of millions of women. They may cause, for instance, pain, bleeding and infertility. Fibroids are also the most common reason for a hysterectomy; ...
Jun 7, 2013
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(Medical Xpress)—Researchers at King's College London have for the first time identified a defective gene at the root of Vici syndrome, a rare inherited disorder which affects infants from birth, leading to impaired development ...
Dec 12, 2012
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Screening for mutations influencing the migration of nerve cells in mice, scientists found a gene that plays a role in the transport of proteins within nerve cells. If less of the protein is present in the developing mouse, ...
Feb 1, 2018
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U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging ...
Dec 19, 2017
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Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers ...
Mar 21, 2017
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When neurobiologist David Corey showed up at a rare disease conference in 2017, he had no idea that he would enter a race against time to develop a treatment for it.
Oct 19, 2023
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Two to three percent of the children are born with an intellectual disability. Possibly by a genetic defect, but in 80% of these cases, we do not know – yet - which genes are responsible. VIB researchers at KU Leuven show ...
Aug 31, 2012
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