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3’ UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates

Vector-mediated mutagenesis remains a major safety concern for many gene therapy clinical protocols. Indeed, lentiviral-based gene therapy treatments of hematologic disease can result in oligoclonal blood reconstitution in the transduced cell graft. Specifically, clonal expansion of hematopoietic st...

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Detalles Bibliográficos
Autores principales: Bonner, Melissa A., Morales-Hernández, Antonio, Zhou, Sheng, Ma, Zhijun, Condori, Jose, Wang, Yong-Dong, Fatima, Soghra, Palmer, Lance E., Janke, Laura J., Fowler, Stephanie, Sorrentino, Brian P., McKinney-Freeman, Shannon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8181581/
https://www.ncbi.nlm.nih.gov/pubmed/34141824
http://dx.doi.org/10.1016/j.omtm.2021.04.013