ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
Neuroblastoma-targeted nanoparticles entrapping siRNA specifically knockdown ALK.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.
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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.
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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
30 citing papers in PubMed, 64 citations in OpenAlex.
- A humanized anaplastic lymphoma kinase (ALK)-directed antibody-drug conjugate with pyrrolobenzodiazepine payload demonstrates efficacy in ALK-expressing cancers.Nature communications · 2025Article
- Advances in functional lipid nanoparticles: from drug delivery platforms to clinical applications.3 Biotech · 2024Review
- GD2-targeting therapy: a comparative analysis of approaches and promising directions.Frontiers in immunology · 2024Review
- Therapeutic silencing of mTOR by systemically administered siRNA-loaded neutral liposomal nanoparticles inhibits DMBA-induced mammary carcinogenesis.British journal of cancer · 2022Article
- The Pyrazolo[3,4-Biomedicines · 2022Article
- Nanotechnology-Based Diagnostic and Therapeutic Strategies for Neuroblastoma.Frontiers in pharmacology · 2022Review
- A Focus on Regulatory Networks Linking MicroRNAs, Transcription Factors and Target Genes in Neuroblastoma.Cancers · 2021Review
- Cotargeting of miR-126-3p and miR-221-3p inhibits PIK3R2 and PTEN, reducing lung cancer growth and metastasis by blocking AKT and CXCR4 signalling.Molecular oncology · 2021Article
- Dual Targeting CAR-T Cells with Optimal Costimulation and Metabolic Fitness enhance Antitumor Activity and Prevent Escape in Solid Tumors.Nature cancer · 2021Article
- Retinoids Delivery Systems in Cancer: Liposomal Fenretinide for Neuroectodermal-Derived Tumors.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Cell surface Nucleolin represents a novel cellular target for neuroblastoma therapy.Journal of experimental & clinical cancer research : CR · 2021Article
- Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach.International journal of molecular sciences · 2020Review
- Recent advances in siRNA delivery mediated by lipid-based nanoparticles.Advanced drug delivery reviews · 2020Review
- Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery.Advanced materials (Deerfield Beach, Fla.) · 2019Review
- Tumor-targeted Nanoparticle Delivery of HuR siRNA Inhibits Lung Tumor GrowthMolecular cancer therapeutics · 2017Article
- A Proof-of-Concept for Epigenetic Therapy of Tissue Fibrosis: Inhibition of Liver Fibrosis Progression by 3-Deazaneplanocin A.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- New therapeutic strategies in neuroblastoma: combined targeting of a novel tyrosine kinase inhibitor and liposomal siRNAs against ALK.Oncotarget · 2015Article
- Anaplastic lymphoma kinase: Role in cancer and therapy perspective.Cancer biology & therapy · 2015Review
- Rationale and Means to Target Pro-Inflammatory Interleukin-8 (CXCL8) Signaling in Cancer.Pharmaceuticals (Basel, Switzerland) · 2013Article
- New insights into the genetics of neuroblastoma.Molecular diagnosis & therapy · 2013Review
Corrections and comments
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Authors and funding
15 authors at 4 institutions in 2 countries.
Funding
Abstract
RNA interference molecules have some advantages as cancer therapeutics, including a proved efficacy on both wild-type (WT) and mutated transcripts and an extremely high sequence-specificity. The most significant hurdle to be overcome if exogenous small interfering RNAs (siRNA) is to be used therapeutically is the specific, effective, nontoxic delivery of siRNA to its intracellular site of action. At present, human applications are confined almost exclusively to targets within the liver, where the delivery systems naturally accumulate, and extra-hepatic targets remain a challenge. Anaplastic lymphoma kinase (ALK) is a receptor tyrosine kinase that has recently been shown to contribute to the cell growth and progression of human neuroblastoma (NB). We investigated its potential as a therapeutic target in NB by generating anti-GD₂-targeted nanoparticles that carry ALK-directed siRNA, which are specifically and efficiently delivered to GD₂-expressing NB cells. Relative to free ALK-siRNA, anti-GD₂-targeted liposomal formulations of ALK-siRNA had low plasma clearance, increased siRNA stability, and improved binding, uptake, silencing and induction of cell death, and specificity for NB cells. In NB xenografts, intravenous (i.v.) injection of the targeted ALK-siRNA liposomes showed gene-specific antitumor activity with no side effects. ALK-selective siRNA entrapped in anti-GD₂-targeted nanoparticles is a promising new modality for NB treatment.
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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.