Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector.

Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector.

Tay-Sachs and Sandhoff illness are debilitating genetic ailments that have an effect on the central nervous system resulting in neurodegeneration by means of the buildup of GM2 gangliosides. There aren’t any cures for these ailments and coverings don’t alleviate all signs. Hematopoietic stem cell gene remedy gives a promising therapy technique to ship wild sort […]

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