Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease

The safety and efficacy of lentivirus-mediated gene therapy was recently demonstrated in five male patients with Fabry disease—a rare X-linked lysosomal storage disorder caused by GLA gene mutations that result in multiple end-organ complications. To evaluate the risks of clonal dominance and leukem...

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Detalles Bibliográficos
Autores principales: Saleh, Amr H., Rothe, Michael, Barber, Dwayne L., McKillop, William M., Fraser, Graeme, Morel, Chantal F., Schambach, Axel, Auray-Blais, Christiane, West, Michael L., Khan, Aneal, Fowler, Daniel H., Rupar, C. Anthony, Foley, Ronan, Medin, Jeffrey A., Keating, Armand
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9932294/
https://www.ncbi.nlm.nih.gov/pubmed/36816757
http://dx.doi.org/10.1016/j.omtm.2023.01.003

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