A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects. The study, conducted ...
Sep 28, 2023
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Neuronal Ceroid Lipofuscinosis are a group of devastating neurodegenerative lysosomal storage disorders that begin in childhood. Mutations in CLN3 gene lead to a NCL called Batten disease, characterized by the progressive ...
Jul 6, 2023
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Batten disease is a family of 13 rare, genetically distinct conditions. Collectively, they are the most prevalent cause of neurodegenerative disease in children, affecting 1 in 12,500 live births in the U.S. One of the Batten ...
Jun 30, 2020
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Professor Stéphane Lefrançois, a researcher at the Institut National de la Recherche Scientifique (INRS), is working on Batten disease, a neurodegenerative genetic disease that primarily affects children. His research focuses ...
Feb 28, 2020
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An unprecedented case at Boston Children's Hospital shows that it's possible to do something that's never been done before: identify a patient's unique mutation, design a customized drug to bypass it, manufacture and test ...
Oct 10, 2019
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A team led by researchers at Baylor College of Medicine has uncovered an unexpected mechanism that can explain a form of Batten disease called neuronal ceroid lipofuscinosis 8. The findings published in the journal Nature ...
Nov 5, 2018
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OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people—a finding that holds promise for developing new gene therapies to treat Batten ...
Jul 27, 2018
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The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) today announce the availability of a unique and comprehensive collection of juvenile Batten (CLN3) disease induced pluripotent stem (iPS) ...
Oct 4, 2017
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Researchers are working hard to fight childhood dementia and hope a new enzyme replacement therapy can help those unlucky enough to have such a debilitating disease.
Sep 1, 2017
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2017 is supposed to be the year that FDA finally approves a gene therapy. But last week, the company behind the first approved gene therapy in Europe, uniQure.com, announced that it won't "pursue the renewal of marketing ...
May 2, 2017
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