Scientific Research Resource Core
科研资源核心
基本信息
- 批准号:10261406
- 负责人:
- 金额:$ 20.01万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2015
- 资助国家:美国
- 起止时间:2015-09-15 至 2025-08-31
- 项目状态:未结题
- 来源:
- 关键词:AddressAffectBloodCRISPR/Cas technologyCardiac MyocytesCommunitiesDataDiseaseDuchenne muscular dystrophyDystrophinExonsFree WillFundingGenesGenetic DiseasesGenomeGenomicsGoalsGuide RNAHumanIndividualInternationalLearningLinkMediatingModelingMolecularMuscle CellsMuscular DystrophiesMutationMyopathyPatientsProcessProtocols documentationRNA SequencesReagentResearchResearch PersonnelResearch SupportResourcesServicesSiteTechniquesTechnologyTherapeuticTimeTrainingTranscriptUpdateVisitbiobankclinical phenotypedesignefficacy evaluationgene repairgenome editingimprovedin silicoinduced pluripotent stem cellmutantnext generation sequencingnovelnovel strategiesrepair strategyrestorationtooltraining opportunityweb pageweb site
项目摘要
Project Summary
The breakthrough technology of CRISPR/Cas9-mediated genomic editing offers an entirely new
and powerful therapeutic potential for patients suffering from genetic diseases like Duchenne
Muscular Dystrophy (DMD). Since joining the Wellstone Network in 2015, the UT Southwestern
Myoediting Core has served both as an essential coordinating hub for the Wellstone research
groups (Projects 1 & 2) at UT Southwestern and as a conduit for sharing expertise and resources
with the broader scientific community. Moving forward, our goals for this Shared Research
Resource Core are: (Aim #1) To continue to support Projects 1 and 2 of the UT Southwestern
Wellstone Center by providing the cutting-edge technologies of human iPSCs, CRISPR/Cas9
genomic editing, and cardiomyocyte differentiation. (Aim #2) To help advance the study of
muscular dystrophy worldwide by providing a DMD/BMD Biorepository of patient-derived DMD
iPSCs and their corresponding CRISPR/Cas9-corrected isolates along with information regarding
the clinical phenotype and genomic sequence of the donor patient. (Aim #3) To help other
investigators take full advantage of these resources by freely sharing our streamlined protocols
and providing training (either on site, or in silico) in best practices and approaches. These
resources are available to investigators from other Wellstone Centers as well as the general
scientific community at large.
项目摘要
CRISPR/Cas9介导的基因组编辑的突破性技术提供了一种全新的
对杜氏症等遗传病患者具有强大的治疗潜力
肌肉萎缩症(DMD)。自2015年加入Wellstone网络以来,UT西南
Myoediting Core既是Wellstone研究的重要协调中心,
小组(项目1和2)在UT西南和作为一个管道,分享专业知识和资源
更广泛的科学界。展望未来,我们对这项共享研究的目标
资源核心是:(目标#1)继续支持UT西南的项目1和2
Wellstone中心通过提供人类iPSCs的尖端技术,CRISPR/Cas9
基因组编辑和心肌细胞分化。(Aim#2)帮助推进研究
通过提供患者源性DMD的DMD/BMD生物储存库,
iPSC及其相应的CRISPR/Cas9校正的分离物沿着有关于
供体患者的临床表型和基因组序列。(Aim#3帮助他人
调查人员充分利用这些资源,免费共享我们的简化协议
提供最佳做法和方法方面的培训(现场或计算机模拟)。这些
资源可供调查人员从其他韦尔斯通中心以及一般
整个科学界。
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('RHONDA BASSEL-DUBY', 18)}}的其他基金
Transcriptional Control of Neonatal Heart Regeneration
新生儿心脏再生的转录控制
- 批准号:
10534778 - 财政年份:2021
- 资助金额:
$ 20.01万 - 项目类别:
Transcriptional Control of Neonatal Heart Regeneration
新生儿心脏再生的转录控制
- 批准号:
10365703 - 财政年份:2021
- 资助金额:
$ 20.01万 - 项目类别:
Chemically assisted remodeling of infarcted heart tissue by targeting Wnt lipidation
通过靶向 Wnt 脂化化学辅助重塑梗塞心脏组织
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9364733 - 财政年份:2017
- 资助金额:
$ 20.01万 - 项目类别:
Deciphering the role of a novel micropeptide in cardiac function and dysfunction
破译新型微肽在心脏功能和功能障碍中的作用
- 批准号:
10331296 - 财政年份:2015
- 资助金额:
$ 20.01万 - 项目类别:
Molecular Dissection of Myoblast Fusion In Muscle Development and Regeneration
肌肉发育和再生中成肌细胞融合的分子解剖
- 批准号:
9301471 - 财政年份:2015
- 资助金额:
$ 20.01万 - 项目类别:
Deciphering the role of a novel micropeptide in cardiac function and dysfunction
破译新型微肽在心脏功能和功能障碍中的作用
- 批准号:
10089466 - 财政年份:2015
- 资助金额:
$ 20.01万 - 项目类别:
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