Nature子刊：饶书权/魏妥/程涛/姚瑶合作开发新型LNP，实现体内持久编辑造血干细胞
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TL;DR - Researchers engineered CD34-targeted lipid nanoparticles to deliver CRISPR components into human hematopoietic stem/progenitor cells, enabling durable in vivo editing in humanized mice. The approach could support treatments for inherited blood disorders while preserving long-term hematopoietic function.
- Anti-CD34-conjugated CD34/LNP_DP efficiently delivered mRNA to human CD34+ cells in vitro and in vivo.
- Editing the BCL11A erythroid enhancer sustained fetal hemoglobin expression, relevant to β-thalassemia and sickle cell disease.
- Targeting ELANE exon 2 partially restored impaired neutrophil development in a neutropenia mouse model.
- Intrafemoral delivery achieved lasting edits without disrupting hematopoiesis.
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Nature子刊：饶书权/魏妥/程涛/姚瑶合作开发新型LNP，实现体内持久编辑造血干细胞
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TL;DR - Researchers engineered CD34-targeted lipid nanoparticles to deliver CRISPR components into human hematopoietic stem/progenitor cells, enabling durable in vivo editing in humanized mice. The approach could support treatments for inherited blood disorders while preserving long-term hematopoietic function.
- Anti-CD34-conjugated CD34/LNP_DP efficiently delivered mRNA to human CD34+ cells in vitro and in vivo.
- Editing the BCL11A erythroid enhancer sustained fetal hemoglobin expression, relevant to β-thalassemia and sickle cell disease.
- Targeting ELANE exon 2 partially restored impaired neutrophil development in a neutropenia mouse model.
- Intrafemoral delivery achieved lasting edits without disrupting hematopoiesis.