Gene therapy may be the best hope for curing retinitis pigmentosa (RP), an inherited condition that usually leads to severe vision loss and blinds 1.5 million people worldwide.
Mar 21, 2024
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Their stories are divided into before and after. First, those long years of pain which flooded every moment—school, relationships, work.
Mar 18, 2024
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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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Ophthalmologists at UC Davis Health used an experimental gene therapy last month to treat a patient with wet age-related macular degeneration, or wet AMD. It was the first time the UC Davis Eye Center had used gene therapy.
Aug 29, 2023
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Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the enzyme UGT1A1 which is responsible for metabolizing it into a substance that ...
Aug 17, 2023
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An experimental gene therapy that's applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease.
Dec 15, 2022
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Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman ...
Oct 10, 2022
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Children in Rochester were recently among the first in the nation to receive an experimental treatment for Duchenne muscular dystrophy (DMD). The study is part of an accelerating trend of clinical trials involving gene therapies ...
Feb 10, 2022
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In 2010, doctors treated Doug Olson's leukemia with an experimental gene therapy that transformed some of his blood cells into cancer killers. More than a decade later, there's no sign of cancer in his body.
Feb 6, 2022
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Jordan Janz knew his gamble on an experimental gene therapy for his rare disease might be paying off when he returned to work and a friend sniffed him.
Jun 2, 2021
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