Genetic cause of rare childhood immune disorders discovered
Scientists have pinpointed genetic changes that can leave children born with little to no immune defense against infection.
May 24, 2024
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Scientists have pinpointed genetic changes that can leave children born with little to no immune defense against infection.
May 24, 2024
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Researchers from Nijmegen and Newcastle have discovered a new genetic mutation leading to severe combined immune deficiency disorder (SCID). It's the first time a mutation in the proteasome, a molecular shredder, has been ...
Mar 19, 2024
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An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves ...
May 11, 2021
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A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the 1970s, is treatable ...
Dec 18, 2017
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(HealthDay)—A new approach to gene therapy can restore immune cell types in infants with newly diagnosed X-linked severe combined immunodeficiency (X-SCID), according to a study presented at the annual meeting of the American ...
Dec 11, 2017
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UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year ...
Mar 28, 2017
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While many insights are gained into diseases and genetic disorders from rodent models, there is a pressing need to find models that can more accurately represent disease progression in the human body, such as non-human primates. ...
Dec 27, 2016
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Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare inherited disorder that primarily affects males, scientists from the National ...
Dec 6, 2015
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(HealthDay)—The first test to screen for Severe Combined Immunodeficiency (SCID) in newborns has been approved by the U.S. Food and Drug Administration.
Dec 16, 2014
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Researchers have found that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited ...
Oct 8, 2014
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