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ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013

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

Ana Sergijenko et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 23748415