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Abstract
Antibody-engineered lipid nanoparticles achieved efficient and durable in vivo editing of hematopoietic stem cells in humanized mice.
- The study addresses manufacturing challenges associated with traditional ex vivo gene therapy for hematopoietic stem cells.
- This approach may provide an effective method for treating genetic blood diseases.
- Editing was shown to be both efficient and durable, indicating potential long-term benefits.
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