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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...
Autores principales: | , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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Materias: | |
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 |