ArticleAnnals of hematology2019
Lipid nanoparticle-mediated siRNA delivery for safe targeting of human CML in vivo.
Article in Annals of hematology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 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
47 citing papers in PubMed, 95 citations in OpenAlex.
- Delivery of Monomethyl Auristatin E Using Ionizable Lipid Nanoparticles for B‑Cell Acute Lymphoblastic Leukemia Treatment.ACS omega · 2026Article
- Lipid Nanoparticle Surface Engineering with Heparosan Polysaccharides for Safe and Effective mRNA DeliveryACS applied materials & interfaces · 2026Article
- Combined PD-1 and TIGIT blockade via siRNA enhances anti-leukemic T-cell function and promotes AML cell apoptosis.Scientific reports · 2026Article
- Review
- Reflecting on 30 years of miRNA biology in malignant hematology: current challenges and future directions.Blood advances · 2025Review
- Emerging lipid nanoparticle systems capable of efficient intramuscular RNA delivery.Nanomedicine (London, England) · 2025Review
- RNA activation ofMolecular therapy. Nucleic acids · 2025Article
- Synthetic carbon-based lanthanide upconversion nanoparticles for enhanced photothermal therapy.Nature communications · 2025Article
- 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
- Article
- Nanocarriers: Exploring the Potential of Oligonucleotide Delivery.Current drug delivery · 2025Review
- Incorporating mRNA therapeutics into biological treatments of hematologic malignancies.Frontiers in immunology · 2025Review
- mRNA vaccine development and applications: A special focus on tumors (Review).International journal of oncology · 2024Review
- Advancements and Future Prospects in Molecular Targeted and siRNA Therapies for Chronic Myeloid Leukemia.Biomolecules · 2024Review
- Targeting ferroptosis for leukemia therapy: exploring novel strategies from its mechanisms and role in leukemia based on nanotechnology.European journal of medical research · 2024Review
- Article
- Nanomaterial-Based Strategies for Attenuating T-Cell-Mediated Immunodepression in Stroke Patients: Advancing Research Perspectives.International journal of nanomedicine · 2024Review
- RNA-based translation activators for targeted gene upregulation.Nature communications · 2023Article
- Article
- Article
Corrections and comments
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Authors and funding
20 authors at 5 institutions in 2 countries.
Funding
Abstract
Efficient and safe delivery of siRNA in vivo is the biggest roadblock to clinical translation of RNA interference (RNAi)-based therapeutics. To date, lipid nanoparticles (LNPs) have shown efficient delivery of siRNA to the liver; however, delivery to other organs, especially hematopoietic tissues still remains a challenge. We developed DLin-MC3-DMA lipid-based LNP-siRNA formulations for systemic delivery against a driver oncogene to target human chronic myeloid leukemia (CML) cells in vivo. A microfluidic mixing technology was used to obtain reproducible ionizable cationic LNPs loaded with siRNA molecules targeting the BCR-ABL fusion oncogene found in CML. We show a highly efficient and non-toxic delivery of siRNA in vitro and in vivo with nearly 100% uptake of LNP-siRNA formulations in bone marrow of a leukemic model. By targeting the BCR-ABL fusion oncogene, we show a reduction of leukemic burden in our myeloid leukemia mouse model and demonstrate reduced disease burden in mice treated with LNP-BCR-ABL siRNA as compared with LNP-CTRL siRNA. Our study provides proof-of-principle that fusion oncogene specific RNAi therapeutics can be exploited against leukemic cells and promise novel treatment options for leukemia patients.
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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.