A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those ...
Jul 17, 2024
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In biomedical research involving human subjects, research ethics committees around the world have traditionally emphasized individual rights and protections for participants, including informed consent. The emerging field ...
Jul 12, 2024
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Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can trigger innate immune pathways, leading to the initiation of adaptive immune responses. A new review article published in the journal Human Gene ...
Jul 10, 2024
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Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades. But decades can feel ...
Jun 27, 2024
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A viral gene therapy developed by University at Buffalo researchers has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...
Jun 13, 2024
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In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross ...
May 16, 2024
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Breast cancer is the second-most common cancer diagnosis for U.S. women, and the second-leading cause of female cancer deaths. In recent years, breast cancer treatments have improved significantly, thanks to targeted gene ...
Apr 24, 2024
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In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, ...
Mar 14, 2024
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Recent clinical trials utilizing high doses of adeno-associated virus (AAV) vectors have highlighted a new challenge to AAV gene transfer—activation of the complement system. A new article in Human Gene Therapy describes ...
Feb 22, 2024
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Society learned about the value of mRNA during the COVID-19 pandemic when we saw scientists and medical professionals harness its power to deliver a vaccine for the virus within a year.
Feb 20, 2024
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