ArticlePloS one2014
Systemic delivery of siRNA down regulates brain prion protein and ameliorates neuropathology in prion disorder.
Article in PloS one, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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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
16 citing papers in PubMed, 21 citations in OpenAlex.
- The Role of Microemulsions in Enhancing the Stability of Biopharmaceuticals.Drug design, development and therapy · 2026Review
- Therapeutic strategies in prion disease: current evidence, translational challenges, and emerging directions.Frontiers in neuroscience · 2026Review
- Prion protein (PrP) profiles in blood and CSF: insights into pre-symptomatic and symptomatic prion disease.Brain communications · 2026Article
- Therapeutic Trajectories in Human Prion Diseases.Sub-cellular biochemistry · 2025Review
- New Therapeutic Modalities in Prion Diseases.Sub-cellular biochemistry · 2025Review
- Perspectives on CRISPR Genome Editing to Prevent Prion Diseases in High-Risk Individuals.Biomedicines · 2024Article
- Inducing prion protein shedding as a neuroprotective and regenerative approach in pathological conditions of the brain: from theory to facts.Neural regeneration research · 2023Review
- Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints.Nucleic acids research · 2020Article
- The Role of Vesicle Trafficking Defects in the Pathogenesis of Prion and Prion-Like Disorders.International journal of molecular sciences · 2020Review
- Development of Artificial Plasma Membranes Derived Nanovesicles Suitable for Drugs Encapsulation.Cells · 2020Article
- Antisense oligonucleotides extend survival of prion-infected mice.JCI insight · 2019Article
- Lithium as a disease-modifying agent for prion diseases.Translational psychiatry · 2018Article
- Inability of DNAzymes to cleave RNA in vivo is due to limited Mg[Formula: see text] concentration in cells.European biophysics journal : EBJ · 2018Article
- BACE1 inhibition by microdose lithium formulation NP03 rescues memory loss and early stage amyloid neuropathology.Translational psychiatry · 2017Article
- Balancing Trained Immunity with Persistent Immune Activation and the Risk of Simian Immunodeficiency Virus Infection in Infant Macaques Vaccinated with Attenuated Mycobacterium tuberculosis or Mycobacterium bovis BCG Vaccine.Clinical and vaccine immunology : CVI · 2017Article
- The P42 peptide and Peptide-based therapies for Huntington's disease.Orphanet journal of rare diseases · 2016Review
Corrections and comments
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Authors and funding
17 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
One of the main challenges for neurodegenerative disorders that are principally incurable is the development of new therapeutic strategies, which raises important medical, scientific and societal issues. Creutzfeldt-Jakob diseases are rare neurodegenerative fatal disorders which today remain incurable. The objective of this study was to evaluate the efficacy of the down-regulation of the prion protein (PrP) expression using siRNA delivered by, a water-in-oil microemulsion, as a therapeutic candidate in a preclinical study. After 12 days rectal mucosa administration of Aonys/PrP-siRNA in mice, we observed a decrease of about 28% of the brain PrP(C) level. The effect of Aonys/PrP-siRNA was then evaluated on prion infected mice. Several mice presented a delay in the incubation and survival time compared to the control groups and a significant impact was observed on astrocyte reaction and neuronal survival in the PrP-siRNA treated groups. These results suggest that a new therapeutic scheme based an innovative delivery system of PrP-siRNA can be envisioned in prion disorders.
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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.