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A Scalable, Antibody-Free Lipid Nanoparticle Achieves Preferential mRNA Delivery to Human HSPCs In Vivo Without Conditioning

Academic source · Molecular therapy : the journal of the American Society of Gene Therapy

Source published: 2026-10-23

Site published: 2026-11-03 · Updated: 2026-11-03

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What the paper reports

A ligand-free nanoparticle formulation preferentially delivered mRNA to human hematopoietic stem and progenitor cells in humanized mice without conditioning. It reduced hepatic delivery relative to an SM-102 comparator and enabled functional Cas9-mediated editing of human CD34-positive cells. Single-cell analyses supported tolerability under the conditions examined. Durable stem-cell function and clinical delivery still require further investigation.

Why this paper is included

Could simplify in vivo hematopoietic gene therapy; humanized-mouse delivery and short-term molecular profiling do not demonstrate durable clinical stem-cell correction.

The interpretation above is analysis, not a conclusion attributed to the original source.

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mRNA / LNP

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