Currently, the condition has no definitive treatments other than liver transplant, but the team shows that in mouse models the new therapy can significantly improve liver health and bile duct development – even after liver damage has already begun.
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This study will make basic research on AAV vectors for gene delivery and preclinical gene therapy studies in mice more efficient and reproducible.
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This work has important implications for the large group of patients with advanced heart failure for whom there are currently no treatments to improve their condition.
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This new technology can effectively regulate gene expression, a promising solution to improve gene therapy clinical applications.
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Nearly 25 years after the discovery that mutations in MECP2 gene cause Rett syndrome, the first gene therapy trial for this difficult-to-treat condition is underway.
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Knocking down the Hippo signaling pathway in pig cardiomyocytes after a heart attack, resulted in heart tissue renewal and improved function.
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More than 30 million people in the United States alone are affected by osteoarthritis (OA), a degenerative joint disease, and that number is expected to
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Francis Collins, NIH Director, and Scott Gottlieb, FDA Commissioner, recently announced plans to eliminate oversight of new gene therapy experiments by the NIH’s Recombinant DNA
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Osteoarthritis is a multifactorial degenerative joint disorder that poses a major challenge for treatment. This condition currently affects 28 million people in the United States
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On April 1, 2016, the European Medicines Agency (EMA) approved the first ex-vivo (outside the body) gene therapy in the Western Hemisphere. Diseased stem cells
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