Models Core
模型核心
基本信息
- 批准号:7907721
- 负责人:
- 金额:$ 35.21万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:
- 资助国家:美国
- 起止时间:至
- 项目状态:未结题
- 来源:
- 关键词:AngiographyAnimal ModelAnimalsAnteriorBlood CirculationBlood VesselsBreedingCaringCathetersCell Membrane PermeabilityCellular biologyChildClinicalDoctor of PhilosophyDoseDuchenne muscular dystrophyDyesDystrophinElementsEvans blue stainExposure toFluorescenceGastrocnemius MuscleGene DeliveryGene ExpressionGenesHousingHumanImmunologyImmunosuppressive AgentsInfantIsometric ExerciseLaboratoriesLower ExtremityMacacaMacaca mulattaMaintenanceMeasurementMeasuresModelingMonkeysMuscleMuscle functionMuscular DystrophiesOhioPediatric HospitalsPerformancePharmaceutical PreparationsPhysiologicalPhysiologyPolypeptide N-acetylgalactosaminyltransferaseProteinsProtocols documentationRecovery of FunctionReporter GenesResearch InstituteResearch PersonnelResistanceRouteServicesSystemTechniquesTestingTimeTransferaseTransgenesTreatment EfficacyUniversitiesValidationVirusclinical applicationfemoral arterygene functiongene therapymdx mousemicro-dystrophinmouse modelneutralizing antibodynonhuman primateresearch studyskillstransgene expressionvector
项目摘要
The Models Core (Core C) will provide four essential support elements for Projects 1 and 2. First, the
Models Core will be responsible for breeding and maintaining mdx mice for experiments described in both
projects 1 and 2. The mdx mouse model for Duchenne muscular dystrophy is essential for verifying gene
therapeutic efficacy as proposed in this U54 application. Second, the Models Core will provide all
physiological assessments of gene function in the mdx mouse. These consist offeree and eccentric
contraction measurements of the extensor digitorum longus muscle. These measures are known to be
impaired in this FDA accepted animal model for DMD, and thus will provide important criteria for each
Project to demonstrate a physiologically significant clinical measure of improvement in muscle function.
Such validation is critical for demonstrating efficacy. Third, the core will be responsible for the housing and
care of rhesus macaque monkeys for experiments described in both projects. Non-human primate animals
are essential to evaluate the immunology, distribution, and efficacy in a larger animal species for review by
the FDA. Fourth, the Core will perform and optimize regional vascular delivery protocols, in both the mdx
mouse and rhesus macaque, to deliver human micro-dystrophin and CT GAINAc transferase via rAAVSmediated
gene delivery to the muscles of the lower limb via the femoral artery. The skills involved in this
delivery system are best acquired by repeated performance. Providing this technique in the Core is
essential to achieve uniformity for both projects, and is essential for consistent distribution of transgenes
within the targeted muscle (gastrocnemius).
模型核心(核心C)将为项目1和2提供四个基本支持要素。一是
Models Core将负责繁殖和维持mdx小鼠,以进行两项研究中所述的实验。
项目1和2。Duchenne型肌营养不良症mdx小鼠模型是验证基因的必要条件
如该U54申请中所提出的治疗效果。其次,模型核心将提供所有
mdx小鼠中基因功能的生理学评估。这些包括offeree和偏心
趾长伸肌的收缩测量。这些措施被认为是
在这个FDA接受的DMD动物模型中受损,因此将为每种疾病提供重要标准。
项目旨在证明肌肉功能改善的生理学显著临床措施。
这种验证对于证明功效至关重要。第三,核心将负责住房和
在这两个项目中描述的实验中照顾恒河猴。手术的非人灵长类动物
对于评价较大动物种属的免疫学、分布和疗效至关重要,
FDA。第四,Core将在mdx和mdx中执行和优化区域血管输送方案。
小鼠和恒河猴,以通过rAAV介导的递送人微肌养蛋白和CT GAINAc转移酶
通过股动脉将基因输送到下肢的肌肉。这其中所涉及的技能
传递系统最好通过重复的性能获得。在核心中提供这种技术是
这对两个项目实现一致性至关重要,对转基因的一致分布至关重要。
在目标肌肉(腓肠肌)内。
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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{{ truncateString('LOUIS G CHICOINE', 18)}}的其他基金
Mechanisms of PRMT regulation of pulmonary endothelial cell function
PRMT调节肺内皮细胞功能的机制
- 批准号:
9130380 - 财政年份:2015
- 资助金额:
$ 35.21万 - 项目类别:
Pulmonary Vascular Targeting of Gene Delivery Using Directed Evolution of AAV
利用 AAV 定向进化进行肺血管基因递送靶向
- 批准号:
7455860 - 财政年份:2007
- 资助金额:
$ 35.21万 - 项目类别:
Pulmonary Vascular Targeting of Gene Delivery Using Directed Evolution of AAV
利用 AAV 定向进化进行肺血管基因递送靶向
- 批准号:
7315308 - 财政年份:2007
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
6183176 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
6788676 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
6536564 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
6388543 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
2878912 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
INOS GENE TRANSFECTION IN PULMONARY HYPERTENSION
INOS 基因转染治疗肺动脉高压
- 批准号:
6638102 - 财政年份:1999
- 资助金额:
$ 35.21万 - 项目类别:
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