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Rhesus BCL11A enhancer editing to induce fetal globin expression

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NIAID Data Ecosystem2026-03-12 收录
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https://www.ncbi.nlm.nih.gov/sra/SRP276231
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资源简介:
sgRNA ribonucleoprotein complex (RNP) consisting of a single guide RNA (sgRNA) targeting a GATA1 binding site at the BCL11A +58 erythroid enhancer and Cas9 protein was electroporated into rhesus hematopoietic stem/progenitor cells (HSPCs), followed by autologous infusion into rhesus monkeys after myeloablation. Granulocyte and lymphocyte fractions from rhesus peripheral blood and bone marrow samples were collected to evaluate indel frequencies post-transplantation.
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2021-01-21
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