With an approval likely at the EMA, ex vivo gene therapy in hematopoietic stem cells appears poised for prime time.
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Acid ceramidase deficiency: Farber disease and SMA-PME
Orphanet Journal of Rare Diseases Open Access 20 July 2018
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Scott, C., DeFrancesco, L. Gene therapy's out-of-body experience. Nat Biotechnol 34, 600–607 (2016). https://doi.org/10.1038/nbt.3592
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DOI: https://doi.org/10.1038/nbt.3592
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Acid ceramidase deficiency: Farber disease and SMA-PME
Orphanet Journal of Rare Diseases (2018)