VEGF-Mimetic Supramolecular Nanoparticles for Treating Spinocerebellar Ataxia Type 1
VEGF 模拟超分子纳米颗粒用于治疗 1 型脊髓小脑共济失调
基本信息
- 批准号:10578485
- 负责人:
- 金额:$ 36.13万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2023
- 资助国家:美国
- 起止时间:2023-08-01 至 2025-07-31
- 项目状态:未结题
- 来源:
- 关键词:AblationAffectAutopsyBehavioralBenchmarkingBiological AssayBrainBrain StemCAG repeatCentral Nervous SystemCerebellar AtaxiaCerebellar degenerationCerebellumCerebrospinal FluidChemistryClinicConfocal MicroscopyDiseaseDisease modelDrug KineticsElementsEndothelial CellsEndothelial Growth Factors ReceptorEngineeringEnvironmentExhibitsFormulationFreedomGadoliniumGlutamineGoalsHealthHippocampusHistologicHumanIn VitroInductively Coupled Plasma Mass SpectrometryInflammationInheritedInjectionsIntellectual PropertyKnock-in MouseLabelLengthMagnetic Resonance ImagingMeasuresMedicineMetalsMethodsMicroscopyModelingMonitorMotorMusNanostructuresNatureNerve DegenerationNeurodegenerative DisordersNeuronsPathologicPatientsPenetrationPhasePhenotypePhysiologicalPre-Clinical ModelProcessPropertyProteinsRecombinant Vascular Endothelial Growth FactorRepressionResolutionSamplingSpatial DistributionSpinal CordSymptomsTechniquesTechnologyTestingTherapeuticTherapeutic AgentsTimeToxic effectTranslatingTrinucleotide Repeat ExpansionTumorigenicityType 1 Spinocerebellar AtaxiaVEGFA geneVascular Endothelial Growth FactorsVisualizationWorkangiogenesisaqueousataxin-1autosomecomparative efficacycostcytokinedesigndisease phenotypeefficacy evaluationexperienceexperimental studygain of functionhuman modelimmunogenicimprovedin vivolaser spectrometrymanufacturemiddle agemimeticsmouse modelmutantnanonanoparticlenanotherapyneurovascularnovelparticlepeptide amphiphilespeptidomimeticspolyglutaminetreatment strategy
项目摘要
Spinocerebellar ataxia type 1 (SCA1) is a progressive neurodegenerative disease caused by a
polyglutamine expansion in the protein ATXN1. In the course of delineating early mechanisms
underlying neurodegeneration, we made the unexpected discovery that ATXN1 directly regulates the
expression of the angiogenic and neurotrophic cytokine VEGF and that VEGF levels are abnormally
low in the SCA1 mouse brain with pathologic consequences. We have since discovered that delivering
recombinant VEGF is therapeutic in the well-characterized SCA1 knock-in mouse (SCA1154Q/2Q;
Q=glutamine), the most precise existing mouse model of SCA1. However, there are several challenges
with developing recombinant VEGF as a therapy. Here we propose to build upon our recent work
developing a VEGF-mimetic particle that could be readily synthesized with a greater potential to
engage VEGF receptors in both neurons and endothelial cells to maintain neurovascular health in
SCA1. The goal is to engineer a first-in-class nanotherapy for SCA1.
脊髓小脑性共济失调1型(SCA1)是一种进行性神经退行性疾病
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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Puneet Opal其他文献
Puneet Opal的其他文献
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{{ truncateString('Puneet Opal', 18)}}的其他基金
Equipment Supplement: Understanding the Cellular Basis of Movement Disorders
设备补充:了解运动障碍的细胞基础
- 批准号:
10755946 - 财政年份:2023
- 资助金额:
$ 36.13万 - 项目类别:
Elucidating cellular mechanisms underlying neurodegeneration
阐明神经变性的细胞机制
- 批准号:
10647869 - 财政年份:2022
- 资助金额:
$ 36.13万 - 项目类别:
Elucidating cellular mechanisms underlying neurodegeneration
阐明神经变性的细胞机制
- 批准号:
10435954 - 财政年份:2022
- 资助金额:
$ 36.13万 - 项目类别:
Developing novel treatment strategies for Spinocerebellar ataxia type 1
开发 1 型脊髓小脑共济失调的新治疗策略
- 批准号:
9226821 - 财政年份:2016
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the cellular basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
8876831 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the cellular basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
8631893 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the Cellular Basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
10630308 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the Cellular Basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
10403448 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the cellular basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
8719191 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
Understanding the Cellular Basis of Movement Disorders
了解运动障碍的细胞基础
- 批准号:
10160963 - 财政年份:2013
- 资助金额:
$ 36.13万 - 项目类别:
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