Genetic Engineering and Manufacturing of Hematopoietic Stem Cells
作者： Xiuyan Wang , Isabelle Rivière
DOI: 10.1016/J.OMTM.2017.03.003
关键词: Adenosine deaminase deficiency 、 Stem cell 、 Computational biology 、 Primary immunodeficiency 、 Severe combined immunodeficiency 、 Genetically engineered 、 Hematopoietic stem cell transplantation 、 Context (language use) 、 Haematopoiesis 、 Biology 、 Immunology
摘要: The marketing approval of genetically engineered hematopoietic stem cells (HSCs) as the first-line therapy for treatment severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) is a tribute substantial progress that has been made regarding HSC engineering in past decade. Reproducible manufacturing high-quality, clinical-grade, HSCs foundation broadening application this technology. Herein, current state-of-the-art platforms engineer well challenges pertaining production standardization and product characterization are addressed context primary diseases (PIDs) other monogenic disorders.
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