B2M expression

Biomarker

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CAS9 mRNA + B2M-targeting sgrna

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  1. CAS9 mRNA + B2M-targeting sgrna decreases B2M expression in mice.

    “The LNP also enabled functional delivery of Cas9 mRNA and β2M-targeting sgRNA to human CD34+ HSPCs, resulting in efficient gene editing and reduced β2M expression.”

    Animalslipid nanoparticle-delivered Cas9 mRNA and β2M-targeting sgRNAhuman CD34+ hematopoietic stem and progenitor cells

    RNA delivery favored human blood stem cells in mice

    Molecular therapy : the journal of the American Society of Gene Therapy

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Molecular Therapy

RNA delivery favored human blood stem cells in mice

In humanized mice, antibody-free fat particles delivered more messenger RNA to human blood-forming stem and precursor cells than a comparison formulation.In humanized mice, antibody-free fat particles delivered more messenger RNA to human blood-forming stem and precursor cells than a comparison formulation.

Mouse studyInterventions