Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice

Alexander Langford-Smith, Fiona L Wilkinson, Kia J Langford-Smith, Rebecca J Holley, Ana Sergijenko, Steven J Howe, William R Bennett, Simon A Jones, Je Wraith, Catherine Lr Merry, Robert F Wynn, Brian W Bigger

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Medicine & Life Sciences