Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease

Ana Sergijenko, Alexander Langford-Smith, Ai Y Liao, Claire E Pickford, John McDermott, Gabriel Nowinski, Kia J Langford-Smith, Catherine L R Merry, Simon A Jones, J Edmond Wraith, Robert F Wynn, Fiona L Wilkinson, Brian W Bigger

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Medicine & Life Sciences