Preclinical development of DBAS gene therapy
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https://www.ncbi.nlm.nih.gov/sra/SRP527945
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资源简介：
We developed a novel self-inactivating third-generation lentiviral vector encoding RPS19 for Diamond-Blackfan anemia syndrome (DBAS) gene therapy. We optimized the LV design, improved transduction efficiency and assess potential therapeutic efficacy in this study using a novel, biologically relevant, human cellular model of DBAS based on heterozygous disruption of RPS19 in healthy donor CD34+ hematopoietic stem/progenitor cells.
创建时间：
2024-08-23
