Article

In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery

2024-11-01

Abstract excerpt

<h4>SUMMARY</h4> Ex vivo autologous hematopoietic stem cells (HSCs) gene therapy provides promising new treatments for hematological disorders. However, current methods involve complex processes and chemotherapeutic conditioning, leading to limited accessibility for treatment and significant side effects. Here, we developed an antibody-free targeted lipid nanoparticles (LNPs) for mRNA delivery to HSCs in vivo ,...

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Literature Corpus work: 026ea275-9e35-58ae-a858-23c0e3f0bfa2
DOI: 10.1101/2024.10.28.620445

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