ArticleJournal of the American Chemical Society2018
Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution.
Article in Journal of the American Chemical Society, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 76 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
76 citing papers in PubMed, 120 citations in OpenAlex.
- Preparation of targeted lipid nanoparticles for precision nucleic acid delivery.Nature protocols · 2026Review
- Recent Advances in Non-Viral Vectors for Gene Therapy and Gene Delivery: From Lipid Nanoparticles to Engineered Extracellular Vesicles.Pharmaceutics · 2026Review
- Machine learning-enhanced nano-QSAR and multiscale modeling for predictive nanomedicine: applications in herbal therapeutics and neglected tropical diseases.Discover nano · 2026Review
- Engineering platelets as cancer therapeutics.Nature reviews. Clinical oncology · 2026Review
- Lipid nanoparticles for bone marrow-targeted RNA therapeutics.Journal of nanobiotechnology · 2026Review
- Investigating the functional contributions of phospholipids in selective organ targeting lipid nanoparticles.Biomaterials · 2026Article
- Multiplexed lipid nanoparticle barcoding reveals tissue-dynamic kinetic insights and enriched cellular tropism in hepatic zones.Nature communications · 2026Article
- Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components.Small methods · 2026Review
- Impact of Endothelial Diversity and Dysfunction on Cardiovascular Disease.Comprehensive Physiology · 2025Review
- Targeting and tracking mRNA lipid nanoparticles at the particle, transcript and protein level.Nature biomedical engineering · 2025Review
- Membrane-modified lipid nanoparticles for RNA delivery.Molecular therapy. Methods & clinical development · 2025Review
- Microenvironmental determinants of endothelial cell heterogeneity.Nature reviews. Molecular cell biology · 2025Review
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Nature-inspired platform nanotechnology for RNA delivery to myeloid cells and their bone marrow progenitors.Nature nanotechnology · 2025Article
- Purine metabolism in bone marrow microenvironment inhibits hematopoietic stem cell differentiation under microgravity.Stem cell research & therapy · 2025Article
- Barcoded screening identifies nanocarriers for protein delivery to kidney.Nature communications · 2025Article
- Precision Nanomedicine for Cancer: Innovations, Strategies, and Translational Challenges.OncoTargets and therapy · 2025Review
- Navigating the intricate in-vivo journey of lipid nanoparticles tailored for the targeted delivery of RNA therapeutics: a quality-by-design approach.Journal of nanobiotechnology · 2024Review
- Tuning Lipid Nanoparticles for RNA Delivery to Extrahepatic Organs.Advanced materials (Deerfield Beach, Fla.) · 2024Review
- Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing.Small science · 2024Article
16 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Bone marrow endothelial cells (BMECs) regulate their microenvironment, which includes hematopoietic stem cells. This makes BMECs an important target cell type for siRNA or gene editing (e.g., CRISPR) therapies. However, siRNA and sgRNA have not been delivered to BMECs using systemically administered nanoparticles. Given that in vitro nanoparticle screens have not identified nanoparticles with BMEC tropism, we developed a system to quantify how >100 different nanoparticles deliver siRNA in a single mouse. This is the first barcoding system capable of quantifying functional cytosolic siRNA delivery (where the siRNA drug is active), distinguishing it from in vivo screens that quantify biodistribution (where the siRNA drug went). Combining this approach with bioinformatics, we performed in vivo directed evolution, and identified BM1, a lipid nanoparticle (LNP) that delivers siRNA and sgRNA to BMECs. Interestingly, chemical analysis revealed BMEC tropism was not related to LNP size; tropism changed with the structure of poly(ethylene glycol), as well as the presence of cholesterol. These results suggest that significant changes to vascular targeting can be imparted to a LNP by making simple changes to its chemical composition, rather than using active targeting ligands. BM1 is the first nanoparticle to efficiently deliver siRNA and sgRNA to BMECs in vivo, demonstrating that this functional in vivo screen can identify nanoparticles with novel tropism in vivo. More generally, in vivo screening may help reveal the complex relationship between nanoparticle structure and tropism, thereby helping scientists understand how simple chemical changes control nanoparticle targeting.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.