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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In a study published in Communications Medicine, researchers from Tokyo Medical and Dental University (TMDU) have unveiled a groundbreaking genome-editing technique. This innovation holds promise for treating Charcot–Marie–Tooth ...
Dec 19, 2023
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A new study, published online ahead of print in Journal for ImmunoTherapy of Cancer on December 6, has identified a novel biomarker for ICIs in human cancers.
Dec 13, 2023
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A new review article in Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases. The article ...
Nov 13, 2023
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