Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

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Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs…

Continue ReadingOverexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

Development of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis

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Osteoarthritis (OA) is the most prevalent musculoskeletal disease affecting nearly 27 million people in the United States, and is the leading cause of chronic disability in the United States. Current…

Continue ReadingDevelopment of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis