The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy likely died after having a reaction to the virus that delivered the therapy in his body, researchers concluded in an early study.
Jun 1, 2023
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A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, ...
Jun 2, 2022
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By reprogramming tumor cells to become the body's defenders, Filipe Pereira and his colleagues hope to improve current cancer treatments.
Dec 22, 2022
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The first three coronavirus vaccines earned Emergency Use Authorization more than a year ago. To date, no other vaccines have been put into use in the U.S—but that will soon change. More than 40 vaccines are undergoing ...
May 5, 2022
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Two Houston researchers from Baylor College of Medicine and Texas Children's Hospital were part of an international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited ...
Jul 11, 2013
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Scientists at the Howard Hughes Medical Institute's Janelia Research Campus and the University of California, Berkeley have developed a powerful new tool for neuroscientists—a viral vector called rAAV2-retro, which efficiently ...
Oct 7, 2016
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Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform ...
May 18, 2011
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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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Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine.
Jun 27, 2012
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The blood–brain barrier (BBB) is a stringent, nearly impenetrable layer of cells that guards the brain, protecting the vital organ from hazards in the bloodstream such as toxins or bacteria and allowing only a very limited ...
Apr 19, 2023
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