ArticleFrontiers in neuroscience2017
Improving the Delivery of SOD1 Antisense Oligonucleotides to Motor Neurons Using Calcium Phosphate-Lipid Nanoparticles.
Article in Frontiers in neuroscience, 2017. 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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Who cites it
30 citing papers in PubMed, 68 citations in OpenAlex.
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- The effects of the GLP1 analog liraglutide on allodynia and motor coordination in peripheral neuropathy induced by a chemotherapeutic agent, cisplatin.Journal of molecular histology · 2025Article
- Engineering circular RNA for molecular and metabolic reprogramming.Functional & integrative genomics · 2024Review
- Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection.Molecular therapy. Nucleic acids · 2023Article
- Biomedical application of 2D nanomaterials in neuroscience.Journal of nanobiotechnology · 2023Review
- Preparation and application of calcium phosphate nanocarriers in drug delivery.Materials today. Bio · 2022Review
- Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology.Pharmaceutics · 2022Review
- Taking Advantages of Blood-Brain or Spinal Cord Barrier Alterations or Restoring Them to Optimize Therapy in ALS?Journal of personalized medicine · 2022Review
- SOD1 in ALS: Taking Stock in Pathogenic Mechanisms and the Role of Glial and Muscle Cells.Antioxidants (Basel, Switzerland) · 2022Review
- Antioxidant Therapy in Oxidative Stress-Induced Neurodegenerative Diseases: Role of Nanoparticle-Based Drug Delivery Systems in Clinical Translation.Antioxidants (Basel, Switzerland) · 2022Review
- Formulation, Characterization, and Cytotoxicity Evaluation of Lactoferrin Functionalized Lipid Nanoparticles for Riluzole Delivery to the Brain.Pharmaceutics · 2022Article
- Nanocarriers for Delivery of Oligonucleotides to the CNS.International journal of molecular sciences · 2022Review
- Noncoding RNA therapeutics for substance use disorder.Advances in drug and alcohol research · 2022Article
- Research Into the Effect of Proton Pump Inhibitors on Lungs and Leukocytes.The Turkish journal of gastroenterology : the official journal of Turkish Society of Gastroenterology · 2021Article
- A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed?Translational neurodegeneration · 2021Review
- Emerging Applications of Nanotechnology in Healthcare Systems: Grand Challenges and Perspectives.Pharmaceuticals (Basel, Switzerland) · 2021Review
- Zebrafish Models for the Safety and Therapeutic Testing of Nanoparticles with a Focus on Macrophages.Nanomaterials (Basel, Switzerland) · 2021Review
- Targeting circular RNAs as a therapeutic approach: current strategies and challenges.Signal transduction and targeted therapy · 2021Review
- Advances in the Design of (Nano)Formulations for Delivery of Antisense Oligonucleotides and Small Interfering RNA: Focus on the Central Nervous System.Molecular pharmaceutics · 2021Review
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease affecting the upper and lower motor neurons in the motor cortex and spinal cord. Abnormal accumulation of mutant superoxide dismutase I (SOD1) in motor neurons is a pathological hallmark of some forms of the disease. We have shown that the orderly progression of the disease may be explained by misfolded SOD1 cell-to-cell propagation, which is reliant upon its active endogenous synthesis. Reducing the levels of SOD1 is therefore a promising therapeutic approach. Antisense oligonucleotides (ASOs) can efficiently silence proteins with gain-of-function mutations. However, naked ASOs have a short circulation half-life and are unable to cross the blood brain barrier (BBB) warranting the use of a drug carrier for effective delivery. In this study, calcium phosphate lipid coated nanoparticles (CaP-lipid NPs) were developed for delivery of SOD1 ASO to motor neurons. The most promising nanoparticle formulation (Ca/P ratio of 100:1), had a uniform spherical core-shell morphology with an average size of 30 nm, and surface charge (ζ-potential) of -4.86 mV. The encapsulation efficiency of ASO was 48% and stability studies found the particle to be stable over a period of 20 days.
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