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