ArticleScientific reports2021
Neuropeptide Y (NPY) intranasal delivery alleviates Machado-Joseph disease.
Article in Scientific reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 23 citations in OpenAlex.
- Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.Biomarker research · 2026Article
- Intranasal Drug Delivery in Neuropharmacology: Advances in Brain-Targeted Therapies and Bioethical Challenges.Biomedicines · 2026Review
- Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.Research square · 2026Article
- Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.medRxiv : the preprint server for health sciences · 2026Article
- Brain-Bone Axis in Physiological and Pathological Conditions.International journal of molecular sciences · 2025Review
- Restoring neuropetide Y levels in the hypothalamus ameliorates premature aging phenotype in mice.GeroScience · 2025Article
- Intranasal Application of Peptides Modulating the Neuropeptide Y System.ACS pharmacology & translational science · 2025Article
- Neuropeptide Y and Pain: Insights from Brain Research.ACS pharmacology & translational science · 2024Review
- Neuropeptides and Their Roles in the Cerebellum.International journal of molecular sciences · 2024Review
- Intranasal neuropeptide Y1 receptor antagonism improves motor deficits in symptomatic SOD1 ALS mice.Annals of clinical and translational neurology · 2023Article
- Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Nanotechnological Advances for Nose to Brain Delivery of Therapeutics to Improve the Parkinson Therapy.Current neuropharmacology · 2023Review
- Coenzyme Q10 Supplementation Increases Removal of theNutrients · 2022Article
- Neuroprotective Effects of Neuropeptide Y against Neurodegenerative Disease.Current neuropharmacology · 2022Review
- A Variant in Genes of the NPY System as Modifier Factor of Machado-Joseph Disease in the Chinese Population.Frontiers in aging neuroscience · 2022Article
- Differential NPY-Y1 Receptor Density in the Motor Cortex of ALS Patients and Familial Model of ALS.Brain sciences · 2021Article
- The Role of Neuropeptide Y in the Nucleus Accumbens.International journal of molecular sciences · 2021Review
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Machado-Joseph disease (MJD) is the most common dominantly-inherited ataxia worldwide with no effective treatment to prevent, stop or alleviate its progression. Neuropeptide Y (NPY) is a neuroprotective agent widely expressed in the mammalian brain. Our previous work showed that NPY overexpression mediated by stereotaxically-injected viral vectors mitigates motor deficits and neuropathology in MJD mouse models. To pursue a less invasive translational approach, we investigated whether intranasal administration of NPY would alleviate cerebellar neuropathology and motor and balance impairments in a severe MJD transgenic mouse model. For that, a NPY solution was administered into mice nostrils 5 days a week. Upon 8 weeks of treatment, we observed a mitigation of motor and balance impairments through the analysis of mice behavioral tests (rotarod, beam walking, pole and swimming tests). This was in line with a reduction of cerebellar pathology, evidenced by a preservation of cerebellar granular layer and of Purkinje cells and reduction of mutant ataxin-3 aggregate numbers. Furthermore, intranasal administration of NPY did not alter body weight gain, food intake, amount of body fat nor cholesterol or triglycerides levels. Our findings support the translational potential of intranasal infusion of NPY as a pharmacological intervention in MJD.
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