An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
Jan 28, 2021
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A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop ...
Apr 17, 2019
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Growing up in Devon in the 1980s, brothers James and Matt (names have been changed) had to be careful with their antics – a cut or bruise could land them in hospital.
Jan 15, 2019
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Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. In new research presented today, scientists successfully used gene therapy to slow the progression ...
Nov 4, 2018
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Toby Willis gave up on medical miracles long ago.
Apr 10, 2018
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Decades of scientific research into areas including plant genetics and data science helped doctors successfully carry out an experimental therapy to create a new skin for a seven-year-old boy suffering from a rare genetic ...
Dec 14, 2017
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The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie "Lorenzo's Oil."
Oct 4, 2017
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An article published in Experimental Biology and Medicine (Volume 242, Issue 13, July, 2017) reports that gene therapy may be used to as an intermediate therapy for newborns with surfactant protein deficiencies until lung ...
Jul 26, 2017
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Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental ...
Dec 3, 2016
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