Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear ...
Oct 15, 2021
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241
Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients' eyesight and the sensitivity of the retina within weeks of treatment. Both of these ...
May 3, 2015
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The new drug sotorasib reduces tumor size and shows promise in improving survival among patients with lung tumors caused by a specific DNA mutation, according to results of a global phase 2 clinical trial. The drug is designed ...
Jun 4, 2021
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A new study led by the University of Bristol has shown a common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality ...
Apr 21, 2020
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A new publication by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the ...
Sep 9, 2020
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For people diagnosed with advanced pancreatic cancer, the outlook is about as grim as it gets: the average patient won't live longer than a year.
Jun 3, 2019
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An experimental cream-based gene therapy may soon become the first U.S. government-approved means for treating a rare and devastating skin disease that produces "butterfly children."
Mar 29, 2022
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(Medical Xpress) -- British scientists are to carry out the largest trial anywhere in the world of a gene therapy for cystic fibrosis.
Mar 16, 2012
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A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University ...
Jul 12, 2021
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46
A pilot gene therapy treatment for sickle cell disease, restoring patients' ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children's Hospital ...
Jan 10, 2020
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