
DOI . ORG {
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Title[redir]:
Systemic delivery of factor IX messenger RNA for protein replacement therapy | PNAS
Description:
Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA deliver...
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Keywords {🔍}
fix, protein, mrna, hfix, levels, activity, delivery, therapeutic, clotting, fig, animals, lunar, lipid, lnps, liver, hemophilia, mice, factor, crossref, pubmed, google, scholar, rna, treatment, mgkg, reaction, immune, administration, serum, mouse, human, circulating, mrnas, elisa, therapy, nanoparticles, lnp, dose, test, vivo, recombinant, hemophilic, lipids, patients, times, formulation, assay, plasma, gene, normal,
Topics {✒️}
spectra/por flot-a-lyzer ready pe-conjugated streptavidin–biotin complex granulocyte-macrophage colony-stimulating factor supporting information appendix social media facebook granulocyte-colony stimulating factor mrna-based therapeutic options niemann-pick type c2 jan-willem van ittersum 2-distearoyl-sn-glycero-3-phosphocholine immune-mediated hepatocyte destruction lunar-delivered human fix-mrna factor ix-deficient plasma macrophage inflammatory protein-1β log–log standard curve 2-kb xhoi–bstbi fragment factor ix-deficient mice receptor-mediated immune response national academy preclinical evaluation supporting mc3–mrna lnp formulation adenovirus-encapsulated r338a-fix mrna-lunar lnp formulation lunar-mrna lnp formulation methods synthesis fix-deficient mouse model early-stage clinical trials mrna therapeutics-based approach sophisticated auditory–motor learning citation information ast/alt/alp assays proprietary arcturus therapeutics plasma-derived fix protein ga-fix-ap lunar-encapsulated hfix mrna fix-deficient hemophilic mice human gene therapy amounts significantly higher librarian recommend pnas factor ix activity protein replacement therapies lunar-delivered fix mrna figures share pnas highlights newsletter fvii sirna gave hepatic gene silencing lnp delivery platform lunar-hfix mrna lnps good manufacturing practice
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- P Petrini, What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B?
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