Myoediting of Duchenne Muscular Dystrophy
杜氏肌营养不良症的肌编辑
基本信息
- 批准号:10261402
- 负责人:
- 金额:$ 160.58万
- 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:AddressAnimal ModelAwarenessBasic ScienceBecker Muscular DystrophyBiologyBiotechnologyBloodBostonCRISPR/Cas technologyCardiacCardiac MyocytesCardiomyopathiesCaregiversCell LineChildClinicClinicalClinical DataClinical ResearchClinical ServicesCoinCommunitiesCommunity OutreachComplementDNA Sequence AlterationDedicationsDevelopmentDisciplineDiseaseDuchenne muscular dystrophyDystrophinEducationEducation and OutreachEnsureFamilyFoundationsGenerationsGenetic MarkersGenomeGoalsGuide RNAHumanInstitutionKnowledgeMediatingMedical centerMedicineMissionModalityModelingMuscleMuscular DystrophiesNeuromuscular DiseasesPatientsPhenotypePublicationsResearchResearch PersonnelResearch Project GrantsResourcesSamplingSchoolsScienceScientistSeriesSourceSystemTechnologyTherapeuticTrainingTranslatingTranslational ResearchUniversity HospitalsVisionWorkbasebench to bedsidebiobankbiomedical scientistclinical careclinically relevantcollaborative environmentdesigndystrophinopathyeducation resourceseducational atmosphereexon skippinggenome editingimprovedinduced pluripotent stem cellinnovationinsightlecturesmemberneuromuscularnew technologynext generationnovel therapeutic interventionnovel therapeuticspatient advocacy grouppatient outreachpreclinical studyprogramsrecruitregenerativeresearch studystandard of caretooltranslational study
项目摘要
Project Summary/Abstract for the Overall Component
The techniques of CRISPR/Cas9-mediated genomic editing and the ability to generate induced pluripotent stem
cells (iPSCs) from a sample of a patient’s blood have placed medicine on the brink of a revolution in our ability
to treat, and perhaps even cure, a broad range of genome-based diseases. The overall goal of the UT
Southwestern Wellstone Muscular Dystrophy Specialized Research Center is to improve the treatment provided
to Duchenne muscular dystrophy (DMD) patients by developing a new therapeutic strategy called “myoediting”.
The Center has been built around five integral components. These include: two inter-related research projects
(1) one that will work to optimize the tools for application of CRISPR/Cas9-mediated DMD exon skipping to
permanently restore dystrophin function, and the other (2) that will identify genetic and biomarker associations
with cardiac phenotypes in patients with dystrophinopathies [i.e. DMD and Becker muscular dystrophy (BMD)]
and serve as a primary source for human iPSCs. These projects will be complemented and supported by three
Cores (A) an Administrative Core, that will also direct patient outreach and education, (B) a Myoediting Scientific
Research Resource Core, which will generate DMD/BMD iPSCs from DMD/BMD patients and differentiate them
into iPSC-derived cardiomyocytes as well as house the DMD/BMD Biobank, which will store relevant clinical
data as well as validated guide RNAs for each genetic mutation, and (C) a Training Core, which will enhance the
educational environment in order to recruit, train, and maintain the next generation of transformative investigators
focused on addressing the challenges of muscular dystrophy. We firmly believe myoediting will offer an
innovative therapeutic modality for the treatment of many thousands of DMD patients and offer a long awaited
hope to these patients and their families devastated by DMD.
整体组件的项目摘要/摘要
CRISPR/Cas9介导的基因组编辑技术及其诱导多能干细胞的产生能力
病人血液样本中的细胞(IPSCs)将医学置于我们能力革命的边缘
来治疗,甚至治愈一系列基于基因组的疾病。UT的总体目标
西南韦尔斯通肌营养不良专科研究中心是为提高治疗水平提供的
Duchenne肌营养不良症(DMD)患者通过开发一种名为“肌肉编辑”的新治疗策略。
该中心围绕五个组成部分而建。其中包括:两个相互关联的研究项目
(1)致力于优化CRISPR/Cas9介导的DMD外显子跳过应用的工具
永久性地恢复营养不良蛋白的功能,以及其他(2)将确定遗传和生物标记物关联的
营养不良症患者的心脏表型[即DMD和Becker肌营养不良症(BMD)]
是人类IPSCs的主要来源。这些项目将得到三个项目的补充和支持
核心(A)管理核心,还将指导患者外展和教育,(B)Myoediting Science
研究资源核心,它将从DMD/BMD患者中生成DMD/BMD IPSCs并将其区分开来
转化为IPSC来源的心肌细胞以及容纳DMD/BMD生物库,该生物库将存储相关的临床
数据以及每个基因突变的有效指南RNA,以及(C)训练核心,这将增强
教育环境,以招募、培训和保持下一代变革性调查人员
专注于解决肌营养不良症的挑战。我们坚信MyoEditing将提供一种
用于治疗数千名DMD患者的创新治疗模式,并提供了期待已久的
希望这些被DMD摧毁的患者和他们的家人。
项目成果
期刊论文数量(0)
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科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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RHONDA BASSEL-DUBY的其他文献
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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:
$ 160.58万 - 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:
$ 160.58万 - 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
- 批准号:
9364733 - 财政年份:2017
- 资助金额:
$ 160.58万 - 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:
$ 160.58万 - 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:
$ 160.58万 - 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:
$ 160.58万 - 项目类别:
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