ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Discovery of siRNA lipid nanoparticles to transfect suspension leukemia cells and provide in vivo delivery capability.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed.
- One size does not fit all: A comparative framework for optimizing ribonucleic acid transfection across acute myeloid leukemia cell lines.Biology methods & protocols · 2026Article
- Suppressing t(4;11) Acute Leukemia by Lipopolymer Nanoparticle Delivery of siRNA Targeting KMT2A::AFF1 with Enhanced Extrahepatic Delivery.Advanced healthcare materials · 2025Article
- A hybridized mechano-electroporation technique for efficient immune cell engineering.Journal of advanced research · 2024Article
- Lipopolymer/siRNA Nanoparticles Targeting the Signal Transducer and Activator of Transcription 5A Disrupts Proliferation of Acute Lymphoblastic Leukemia.ACS pharmacology & translational science · 2024Article
- CD45 inhibition in myeloid leukaemia cells sensitizes cellular responsiveness to chemotherapy.Annals of hematology · 2024Article
- Branching Ionizable Lipids Can Enhance the Stability, Fusogenicity, and Functional Delivery of mRNA.Small science · 2023Article
- Article
- Small interfering RNA for cancer treatment: overcoming hurdles in delivery.Acta pharmaceutica Sinica. B · 2020Review
- Article
- Simplifying the Chemical Structure of Cationic Lipids for siRNA-Lipid Nanoparticles.ACS medicinal chemistry letters · 2019Article
- Nanomedicines for the Delivery of Biologics.Pharmaceutics · 2019Article
- Strategies, design, and chemistry in siRNA delivery systems.Advanced drug delivery reviews · 2019Review
- Complexes of Pro-Apoptotic siRNAs and Carbosilane Dendrimers: Formation and Effect on Cancer Cells.Pharmaceutics · 2019Article
- A comparison of inverted and upright laser-activated titanium nitride micropyramids for intracellular delivery.Scientific reports · 2018Article
- Intracellular Delivery by Membrane Disruption: Mechanisms, Strategies, and Concepts.Chemical reviews · 2018Review
- CRISPR/Cas9 Genome Editing of Epidermal Growth Factor Receptor Sufficiently Abolished Oncogenicity in Anaplastic Thyroid Cancer.Disease markers · 2018Article
- Drug discovery and therapeutic delivery for the treatment of B and T cell tumors.Advanced drug delivery reviews · 2017Review
- Small-sized, stable lipid nanoparticle for the efficient delivery of siRNA to human immune cell lines.Scientific reports · 2016Article
- Review
- Mechanical oscillations enhance gene delivery into suspended cells.Scientific reports · 2016Article
Corrections and comments
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Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
As a powerful research tool, siRNA's therapeutic and target validation utility with leukemia cells and long-term gene knockdown is severely restricted by the lack of omnipotent, safe, stable, and convenient delivery. Here, we detail our discovery of siRNA-containing lipid nanoparticles (LNPs) able to effectively transfect several leukemia and difficult-to-transfect adherent cell lines also providing in vivo delivery to mouse spleen and bone marrow tissues through tail-vein administration. We disclose a series of novel structurally related lipids accounting for the superior transfection ability, and reveal a correlation between expression of Caveolins and successful transfection. These LNPs, bearing low toxicity and long stability of >6 months, are ideal for continuous long-term dosing. Our discovery represents the first effective siRNA-containing LNPs for leukemia cells, which not only enables high-throughput siRNA screening with leukemia cells and difficult-to-transfect adherent cells but also paves the way for the development of therapeutic siRNA for leukemia treatment.
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Registered trials
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