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debilitating aspects of lysosomal storage diseases.
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Crystal structure of human beta-hexosaminidase B: understanding the molecular basis of Sandhoff and
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delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid
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Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E.
Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I
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