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LONG TERM CORRECTION OF GM2 GANGLIOSIDOSES IN SANDHOFF MICE THROUGH INTRAVENOUS NEONATAL GENE THERAPY, USING A RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING A NEW HEXOSAMINIDASE VARIANT363

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LONG TERM CORRECTION OF GM2 GANGLIOSIDOSES IN SANDHOFF MICE THROUGH INTRAVENOUS NEONATAL GENE THERAPY, USING A RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR EXPRESSING A NEW HEXOSAMINIDASE VARIANT4300143

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