Autologous Transplantation of Lentivector/Acid Ceramidase–Transduced Hematopoietic Cells in Nonhuman Primates

Farber disease is a rare lysosomal storage disorder (LSD) that manifests due to acid ceramidase (AC) deficiencies and ceramide accumulation. We present a preclinical gene therapy study for Farber disease employing a lentiviral vector (LV-huAC/huCD25) in three enzymatically normal nonhuman primates....

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Detalhes bibliográficos
Main Authors: Walia, Jagdeep S., Neschadim, Anton, Lopez-Perez, Orlay, Alayoubi, Abdulfatah, Fan, Xin, Carpentier, Stéphane, Madden, Melissa, Lee, Chyan-Jang, Cheung, Fred, Jaffray, David A., Levade, Thierry, McCart, J. Andrea, Medin, Jeffrey A.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2011
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Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3155125/
https://ncbi.nlm.nih.gov/pubmed/21280983
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.195
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