ArticleMolecular pharmaceutics2015
CD33-Targeted Lipid Nanoparticles (aCD33LNs) for Therapeutic Delivery of GTI-2040 to Acute Myelogenous Leukemia.
Article in Molecular pharmaceutics, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 31 citations in OpenAlex.
- Advances in ligand-targeted nanodelivery systems for leukemia therapy: from single- to dual-ligand strategies.Journal of translational medicine · 2026Review
- Overcoming therapeutic challenges in acute myeloid leukemia: active targeting strategies by nano-drug delivery systems.Journal of translational medicine · 2026Review
- Lipid Nanovesicles in Cancer Treatment: Improving Targeting and Stability of Antisense Oligonucleotides.Drug design, development and therapy · 2025Review
- Loop33 × 123 CAR-T targeting CD33 and CD123 against immune escape in acute myeloid leukemia.Cancer immunology, immunotherapy : CII · 2024Article
- Development of CD33-Targeted Dual Drug-Loaded Nanoparticles for the Treatment of Pediatric Acute Myeloid Leukemia.Biomacromolecules · 2024Article
- Nonviral delivery systems for antisense oligonucleotide therapeutics.Biomaterials research · 2022Review
- Glycans as Targets for Drug Delivery in Cancer.Cancers · 2022Review
- Nanomedicine for Immunotherapy Targeting Hematological Malignancies: Current Approaches and Perspective.Nanomaterials (Basel, Switzerland) · 2021Review
- Article
- Recent Treatment Advances and the Role of Nanotechnology, Combination Products, and Immunotherapy in Changing the Therapeutic Landscape of Acute Myeloid Leukemia.Pharmaceutical research · 2019Review
- Engineering the RNA-Nanobio Interface.Bioengineering (Basel, Switzerland) · 2017Review
- Recent insights in nanotechnology-based drugs and formulations designed for effective anti-cancer therapy.Journal of nanobiotechnology · 2016Review
- Targeting Approaches of Nanomedicines in Acute Myeloid Leukemia.Dose-response : a publication of International Hormesis SocietyReview
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Authors and funding
15 authors at 2 institutions in 2 countries.
Funding
Abstract
CD33-targeted lipid nanoparticles (aCD33LNs) were synthesized for delivery of GTI-2040, an antisense oligonucleotide (ASO) against the R2 subunit of ribonucleotide reductase, to acute myelogenous leukemia (AML). These LNs incorporated a deoxycholate-polyethylenimine (DOC-PEI) conjugate, which has shown significant activity to facilitate oligonucleotide delivery. Anti-CD33 scFv (aCD33) was added as a targeting ligand. The delivery efficiency of this system was investigated both in vitro and in vivo. When cells were treated with aCD33LN/GTI-2040, significant uptake was observed in CD33 positive Kasumi-1 cells. aCD33LNs loaded with GTI-2040 induced significant down-regulation of R2 mRNA and protein levels in AML cells. Moreover, aCD33LN/GTI-2040 showed a 15-fold reduction in the IC50 of antileukemic drug Ara-C in Kasumi-1 cells. In Kasumi-1 xenograft model, aCD33LN/GTI-2040 showed significant R2 downregulation compared to LN/GTI-2040. Furthermore, aCD33LN/GTI-2040 coadministered with Ara-C was shown to be highly effective in tumor growth inhibition and to greatly increase survival time of mice bearing Kasumi-1 xenograft tumors. The conjugate DOC-PEI has shown an ability to include calcein release from lipid nanoparticles, suggesting a potential mechanism contributing to efficient endosome release by DOC-PEI2K. These results indicate that aCD33LNs are a highly effective vehicle for the therapeutic delivery of antisense agents to AML.
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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.