A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of ...
Jan 30, 2024
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Real-time magnetic resonance (MR) imaging during gene therapy in the nervous system provides foundational impact on optimizing the effectiveness, safety and efficiency of gene therapy delivery to the nervous system.
Jan 29, 2024
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A new study co-led by investigators from Mass Eye and Ear, a member of Mass General Brigham, demonstrated the effectiveness of a gene therapy in restoring hearing function for children suffering from hereditary deafness.
Jan 24, 2024
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By exploiting the technology used in COVID-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology's ...
Jan 10, 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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Indiana University School of Medicine researchers have uncovered vital insights regarding a liver trigger that blocks an undesired immune response from gene therapy, surprisingly resulting in activating specific immune cells ...
Dec 7, 2023
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Modulating the activity of a kinase in motor neurons may help mitigate mitochondrial defects and other symptoms of spinal muscular atrophy, offering a new therapeutic avenue for the devastating disease, according to a Northwestern ...
Dec 5, 2023
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Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new ...
Nov 16, 2023
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Gene therapy has great promise for treating genetic diseases, and even for more common diseases such as atherosclerosis (hardening of the arteries). Over the past decade, the gene-editing technology CRISPR (Clustered Regularly ...
Nov 16, 2023
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Neuroscientists at the Faculty of Medicine and Surgery of the Catholic University, Rome, and the Fondazione Policlinico Universitario Agostino Gemelli IRCCS have genetically modified a molecule, the protein LIMK1, which is ...
Nov 15, 2023
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