Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
收藏NIAID Data Ecosystem2026-03-12 收录
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https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE163912
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The pre-clinical efficacy and safety data support the initiation of a phase I/II clinical trial for β-globin gene correction in patient-derived hematopoietic stem cells for the treatment of sickle cell disease. This entry covers wild type and high fidelity Cas9 genome-wide repair outcomes from the HBB locus using LAM-HTGTS.
创建时间：
2021-08-16
