This week, a 40-year-old patient was treated at UNC's Clinical and Translational Research Center (CTRC) with SB-913, an investigational genome editing therapy for individuals with mucopolysaccharidosis type II (MPS II), a ...
Mar 22, 2018
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Bluebird Bio Inc.'s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.
Sep 19, 2022
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A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of ...
Jan 30, 2024
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A pair of genes work together as "master regulators" in cell division to keep the correct number of chromosomes in each daughter cell. Ipl1 and Mps1 work to hook and unhook chromosomes for proper DNA separation. The discovery ...
Jan 31, 2013
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As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist-oncologist ...
May 26, 2016
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A new study identified a set of 140 genes that may help predict enhanced disease-free survival in patients with non-small cell lung cancer (NSCLC) treated with a combination of immunotherapy and low-dose radiation. The results, ...
Feb 23, 2024
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A team of University of Saskatchewan (USask) researchers are exploring the role of a newly identified cell type in cystic fibrosis (CF), which could lead to effective new types of treatment.
Apr 29, 2024
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Researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.
Jun 15, 2024
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Researchers at University of Massachusetts Medical School and Auburn University College of Veterinary Medicine are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.
Jul 18, 2017
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The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing ...
Aug 29, 2014
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