ArticleGene therapy2002
Delivery of unmodified bioactive ribozymes by an RNA-stabilizing polyethylenimine (LMW-PEI) efficiently down-regulates gene expression.
Article in Gene therapy, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed, 97 citations in OpenAlex.
- The efficacy of oligonucleotide-based gene therapeutics in gene silencing.Theranostics · 2026Review
- Applications of emerging extracellular vesicles technologies in the treatment of inflammatory diseases.Frontiers in immunology · 2024Review
- VEGF Overexpression Significantly Increases Nanoparticle-Mediated siRNA Delivery and Target-Gene Downregulation.Pharmaceutics · 2022Article
- Polyethyleneimine-Based Lipopolyplexes as Carriers in Anticancer Gene Therapies.Materials (Basel, Switzerland) · 2021Review
- Nanoparticle-complexed antimiRs for inhibiting tumor growth and metastasis in prostate carcinoma and melanoma.Journal of nanobiotechnology · 2020Article
- Synthetic circular miR-21 RNA decoys enhance tumor suppressor expression and impair tumor growth in mice.NAR cancer · 2020Article
- Nanoparticle-Mediated Delivery towards Advancing Plant Genetic Engineering.Trends in biotechnology · 2018Review
- Molecular design of upconversion nanoparticles for gene delivery.Chemical science · 2017Review
- Evaluation of in vivo antitumor effects of ANT2 shRNA delivered using PEI and ultrasound with microbubbles.Gene therapy · 2015Article
- Article
- Synthesis and characterization of mannosylated pegylated polyethylenimine as a carrier for siRNA.International journal of pharmaceutics · 2012Article
- Targeted CRM197-PEG-PEI/siRNA Complexes for Therapeutic RNAi in Glioblastoma.Pharmaceuticals (Basel, Switzerland) · 2011Article
- Comparison of distribution and activity of nanoparticles with short interfering DNA (Dbait) in various living systems.Cancer gene therapy · 2011Article
- Polyethylenimine-mediated gene delivery to the lung and therapeutic applications.Drug design, development and therapy · 2009Article
- Characterization of the transgene expression generated by branched and linear polyethylenimine-plasmid DNA nanoparticles in vitro and after intraperitoneal injection in vivo.Journal of controlled release : official journal of the Controlled Release Society · 2008Article
- Applications of RNA interference: current state and prospects for siRNA-based strategies in vivo.Applied microbiology and biotechnology · 2007Review
- Cell and tissue targeting of nucleic acids for cancer gene therapy.Pharmaceutical research · 2007Review
- Biophysical and structural characterization of polyethylenimine-mediated siRNA delivery in vitro.Pharmaceutical research · 2006Article
- Enhancement of the efficiency of non-viral gene delivery by application of pulsed magnetic field.Nucleic acids research · 2006Article
- Delivery systems for the direct application of siRNAs to induce RNA interference (RNAi) in vivo.Journal of biomedicine & biotechnology · 2006Article
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
No grant is acknowledged in the PubMed record.
Abstract
The sequence-specific cleavage of RNA molecules through ribozyme targeting is particularly attractive since it allows the effective abrogation of protein expression. So far, however, use of enzymatically active RNA molecules (ribozymes) has, without chemical modification, been severely hampered by ribozyme instability and poor cellular uptake. In this paper, we present a method for protection and cellular delivery of ribozymes by complexation with a low molecular weight polyethylenimine (LMW-PEI). We show that LMW-PEI almost completely stabilizes ribozymes or any RNA against degradation in vitro. Upon their highly efficient cellular uptake, non-toxic LMW-PEI-complexed ribozymes display intracellular bioactivity already at low concentrations as demonstrated by down-regulation of two different genes in different cell lines. In vivo, LMW-PEI-complexed ribozymes were stabilized after intraperitoneal (i.p.) injections, showed prolonged circulation time and intact ribozymes were detected in the subcutaneous (s.c.) tumor mass 60 min after the injection. In addition, i.p. injections of LMW-PEI-complexed ribozymes targeted against the growth factor pleiotrophin (PTN) resulted in marked reduction of s.c. human melanoma tumor growth and of intratumoral PTN levels in a mouse xenograft model. Thus, this paper describes a novel method for exogenous delivery of any bioactive RNA ribozyme in vitro and in vivo without chemical modification.
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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.