Training Core [Parent Title: NOVEL THERAPEUTICS FOR FSHD]

培训核心 [父标题:FSHD 的新颖疗法]

基本信息

项目摘要

PROJECT SUMMARY Facioscapulohumeral muscular dystrophy (FSHD) causes lifelong severe disability and is one of the most prevalent muscular dystrophies, afflicting both children and adults. While major advances in genetics have strongly implicated the inappropriate expression of a powerful transcription factor, DUX4, and its target genes in the degeneration of muscle fibers in FSHD, no protective pharmacologic treatments yet exist for this disease. The University of Massachusetts School of Medicine (UMMS) Wellstone Muscular Dystrophy Cooperative Research Center is a network of collaborative investigators whose research and training programs focus on developing novel and effective therapeutics for FSHD. The long-term objectives are to meet this need through three highly synergistic projects directed toward drug discovery and optimization, supported by our Cores, collaborators and advisors. Specific Center goals are: 1) identifying FSHD disease modifiers through expanded genomic investigations of a large Utah FSHD kindred in Project 1 to discern native gene variants and regulatory pathways that influence the FSHD clinical phenotype; 2) discovering modulators of DUX4 toxicity in Project 2 using the novel Wellstone FSHD cell and animal models and CRISPR-based inhibition approaches to identify gene and regulatory pathway therapeutic targets; 3) optimizing our lead DUX4 RNA therapeutics and DUX4 signaling compounds in Project 3, in collaboration with industry; 4) partnering with FSHD and patient advocacy groups to support and participate in FSHD research and clinical trials; 5) expanding collaborations with industry partners who have tools and experience to develop FSHD therapeutics; and 6) training the next generation of clinician-scientists and translational researchers, who will be the driving force of our Wellstone therapeutic development program. Three Center Cores will support the research and training activities of this Wellstone Center and also the greater FSHD research and patient communities. These include an Administrative Core to facilitate communication between our investigators at all sites and to connect investigators with patient advocacy groups, particularly the FSH Society, so that we may continue to engage and provide education to individuals with FSHD and their families. The Education and Training Core will continue to oversee the research and clinical training of students and fellows. The Resources Core will expand a unique repository of FSHD biomaterials, including DNA, muscle tissues, myogenic primary cells and muscle cell lines derived from biopsies, and iPSC cells derived from patient fibroblasts to support Wellstone and greater FSHD community research. These materials are available to academic and industry groups and have increasingly been used as FSHD models for biomarker and preclinical therapeutic studies. The Resources Core will utilize novel inducible DUX4 mouse and zebrafish models and a xenograft model that will support our proposed preclinical projects and also will share these models with other FSHD research groups to accelerate FSHD therapeutic development.
项目摘要 面肩肱型肌营养不良症(FSHD)导致终身严重残疾,是最常见的 流行的肌肉萎缩症,折磨儿童和成人。虽然遗传学的重大进展 强烈暗示了一个强大的转录因子DUX 4及其靶基因的不适当表达 在FSHD的肌纤维变性中,还没有保护性的药理学治疗存在。 疾病马萨诸塞州大学医学院(UMMS) 合作研究中心是一个合作研究人员的网络,其研究和培训计划 专注于开发FSHD的新的和有效的治疗方法。长期目标是满足这一需要 通过三个针对药物发现和优化的高度协同项目,由我们的 核心,合作者和顾问。具体的中心目标是:1)确定FSHD疾病修饰因子, 在项目1中,对一个大型犹他州FSHD家族进行了扩展的基因组研究,以辨别天然基因变异 和影响FSHD临床表型的调节途径; 2)发现DUX 4的调节剂 使用新型Wellstone FSHD细胞和动物模型以及基于CRISPR的抑制, 确定基因和调控途径治疗靶点的方法; 3)优化我们的前导DUX 4 RNA 项目3中的药物和DUX 4信号化合物,与工业界合作; 4)与 FSHD和患者倡导团体支持和参与FSHD研究和临床试验; 5) 扩大与拥有开发FSHD疗法的工具和经验的行业合作伙伴的合作; 6)培养下一代临床科学家和翻译研究人员,他们将成为推动 我们的Wellstone治疗开发计划的力量。三个中心核心将支持研究, 该Wellstone中心的培训活动以及更大的FSHD研究和患者社区。这些 包括一个管理核心,以促进我们所有研究中心研究者之间的沟通, 研究人员与患者倡导团体,特别是FSH协会,以便我们可以继续参与 并为FSHD患者及其家人提供教育。教育和培训中心将 继续监督学生和研究员的研究和临床培训。核心资源将扩大 FSHD生物材料的独特储存库,包括DNA,肌肉组织,肌原性原代细胞和肌肉 来源于活组织检查的细胞系和来源于患者成纤维细胞的iPSC细胞,以支持Wellstone和 FSHD社区研究。这些材料可供学术和工业团体使用, 越来越多地被用作生物标志物和临床前治疗研究的FSHD模型。的资源 核心将利用新的诱导DUX 4小鼠和斑马鱼模型和异种移植模型,这将支持我们的研究。 提出了临床前项目,并将与其他FSHD研究小组分享这些模型,以加速 FSHD治疗开发。

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)

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

数据更新时间:{{ journalArticles.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ monograph.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ sciAawards.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ conferencePapers.updateTime }}

{{ item.title }}
  • 作者:
    {{ item.author }}

数据更新时间:{{ patent.updateTime }}

CHARLES P. EMERSON其他文献

CHARLES P. EMERSON的其他文献

{{ item.title }}
{{ item.translation_title }}
  • DOI:
    {{ item.doi }}
  • 发表时间:
    {{ item.publish_year }}
  • 期刊:
  • 影响因子:
    {{ item.factor }}
  • 作者:
    {{ item.authors }}
  • 通讯作者:
    {{ item.author }}

{{ truncateString('CHARLES P. EMERSON', 18)}}的其他基金

CONTROL OF MUSCLE PROTEIN SYNTHESIS DURING MYOGENESIS
肌生成过程中肌肉蛋白合成的控制
  • 批准号:
    8051021
  • 财政年份:
    2010
  • 资助金额:
    $ 12.92万
  • 项目类别:
Identification of inhibitors of hedgehog autoprocessing
刺猬自动加工抑制剂的鉴定
  • 批准号:
    8089846
  • 财政年份:
    2009
  • 资助金额:
    $ 12.92万
  • 项目类别:
Biomarkers for Therapy of FSHD (U54)
FSHD 治疗的生物标志物 (U54)
  • 批准号:
    7932575
  • 财政年份:
    2009
  • 资助金额:
    $ 12.92万
  • 项目类别:
CONTROL OF MUSCLE PROTEIN SYNTHESIS DURING MYOGENESIS
肌生成过程中肌肉蛋白合成的控制
  • 批准号:
    7867022
  • 财政年份:
    2009
  • 资助金额:
    $ 12.92万
  • 项目类别:
Administrative Core - Novel Therapeutics for FSHD
行政核心 - FSHD 的新疗法
  • 批准号:
    10197167
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:
Biomarkers for Therapy of FSHD (U54)
FSHD 治疗的生物标志物 (U54)
  • 批准号:
    8881247
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:
Biomarkers for Therapy of FSHD (U54)
FSHD 治疗的生物标志物 (U54)
  • 批准号:
    8661472
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:
FSHD Pre-clinical Development
FSHD临床前开发
  • 批准号:
    10197171
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:
Novel Therapeutics for FSHD - Resources Core - Core C
FSHD 的新疗法 - 资源核心 - 核心 C
  • 批准号:
    10197168
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:
Biomarkers for Therapy of FSHD (U54)
FSHD 治疗的生物标志物 (U54)
  • 批准号:
    8336877
  • 财政年份:
    2008
  • 资助金额:
    $ 12.92万
  • 项目类别:

相似海外基金

Co-designing a lifestyle, stop-vaping intervention for ex-smoking, adult vapers (CLOVER study)
为戒烟的成年电子烟使用者共同设计生活方式、戒烟干预措施(CLOVER 研究)
  • 批准号:
    MR/Z503605/1
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Research Grant
Early Life Antecedents Predicting Adult Daily Affective Reactivity to Stress
早期生活经历预测成人对压力的日常情感反应
  • 批准号:
    2336167
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Standard Grant
RAPID: Affective Mechanisms of Adjustment in Diverse Emerging Adult Student Communities Before, During, and Beyond the COVID-19 Pandemic
RAPID:COVID-19 大流行之前、期间和之后不同新兴成人学生社区的情感调整机制
  • 批准号:
    2402691
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Standard Grant
Migrant Youth and the Sociolegal Construction of Child and Adult Categories
流动青年与儿童和成人类别的社会法律建构
  • 批准号:
    2341428
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Standard Grant
Elucidation of Adult Newt Cells Regulating the ZRS enhancer during Limb Regeneration
阐明成体蝾螈细胞在肢体再生过程中调节 ZRS 增强子
  • 批准号:
    24K12150
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Understanding how platelets mediate new neuron formation in the adult brain
了解血小板如何介导成人大脑中新神经元的形成
  • 批准号:
    DE240100561
  • 财政年份:
    2024
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Discovery Early Career Researcher Award
RUI: Evaluation of Neurotrophic-Like properties of Spaetzle-Toll Signaling in the Developing and Adult Cricket CNS
RUI:评估发育中和成年蟋蟀中枢神经系统中 Spaetzle-Toll 信号传导的神经营养样特性
  • 批准号:
    2230829
  • 财政年份:
    2023
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Standard Grant
Usefulness of a question prompt sheet for onco-fertility in adolescent and young adult patients under 25 years old.
问题提示表对于 25 岁以下青少年和年轻成年患者的肿瘤生育力的有用性。
  • 批准号:
    23K09542
  • 财政年份:
    2023
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Identification of new specific molecules associated with right ventricular dysfunction in adult patients with congenital heart disease
鉴定与成年先天性心脏病患者右心室功能障碍相关的新特异性分子
  • 批准号:
    23K07552
  • 财政年份:
    2023
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
Issue identifications and model developments in transitional care for patients with adult congenital heart disease.
成人先天性心脏病患者过渡护理的问题识别和模型开发。
  • 批准号:
    23K07559
  • 财政年份:
    2023
  • 资助金额:
    $ 12.92万
  • 项目类别:
    Grant-in-Aid for Scientific Research (C)
{{ showInfoDetail.title }}

作者:{{ showInfoDetail.author }}

知道了