Tag: <span>novel gene therapy</span>

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Novel gene therapy could reduce bleeding risk for hemophilia patients
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Novel gene therapy could reduce bleeding risk for hemophilia patients

by University College London Credit: Pixabay/CC0 Public Domain A single gene therapy injection could dramatically reduce the bleeding risk faced by people with hemophilia B, finds a study involving UCL researchers. For the paper, published in the New England Journal of Medicine, experts from UCL, Royal Free Hospital and biotechnology company Freeline Therapeutics trialed and continue...

Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events
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Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events

by  Children’s Hospital of Philadelphia Lindsey A. George, MD, lead study author and attending hematologist at Children’s Hospital of Philadelphia. Credit: Children’s Hospital of Philadelphia A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction—or in some cases complete elimination—of painful and potentially life-threatening...

New research moves novel gene therapy for heart failure closer to the clinic
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New research moves novel gene therapy for heart failure closer to the clinic

by  Baylor College of Medicine A stained section of a pig heart was treated with the viral vector 33 days after injection. Credit: S. Liu et al., Science Translational Medicine (2021) Research at Baylor College of Medicine, the Texas Heart Institute, and collaborating institutions are moving a novel promising gene therapy to treat heart failure closer...