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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells

Posted in biotech/medical, genetics, nanotechnology

The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.