A new study has shown that a commonly used vector for large gene transfer can successfully deliver genes to retinal cells in the laboratory, but when injected subretinally into rats it provokes a robust and acute inflammatory ...
Nov 12, 2019
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Researchers are making great strides toward de-veloping gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement. A review of the approaches currently ...
Oct 29, 2019
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Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was born with a blinding disease. This ushered in a period of hope and progress for the field of gene therapy aimed at curing blindness, ...
Sep 10, 2019
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Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...
Aug 21, 2019
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An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe disease ...
Aug 13, 2019
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It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...
Jul 19, 2019
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A therapeutic gene delivered into the spinal canal of infant rhesus monkeys was still being expressed after nearly 4 years, with no evidence of acute or chronic neuronal toxicity, according to a new study published in Human ...
Jun 17, 2019
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Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem ...
Apr 18, 2019
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When the Human Genome Project was completed, in 2003, it opened the door to a radical new idea of health—that of personalized medicine, in which disease risk and appropriate treatment would be gleaned from one's genetic ...
Mar 25, 2019
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Yale researchers have developed a new way to efficiently engineer immune cells, an advance which enhances the ability to fend off cancer and other diseases, they report in the Feb. 25 issue of the journal Nature Methods.
Feb 26, 2019
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