课题基金 / 基金详情

Translating Pathomechanisms into Treatment for Spinal Muscular Atrophies

Translating Pathomechanisms into Treatment for Spinal Muscular Atrophies
将病理机制转化为脊髓性肌萎缩症的治疗
批准号:
10665141
负责人:
Charlotte Jane Sumner
金额:
$16.38万
依托单位:
依托单位国家:
美国
项目类别:
财政年份:
2022
资助国家:
美国
项目状态:
已结题
起止时间:
2022-07-13 至 2023-04-30

项目摘要

项目成果

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中文摘要
翻译
项目总结
英文摘要
PROJECT SUMMARY Spinal muscular atrophies (SMAs) are monogenetic motor neuron (MN) diseases that cause debilitating muscle weakness and often early mortality. Our research program focuses on advancing therapeutics for two forms of SMA: proximal SMA caused by recessive, loss-of-function mutations of the survival motor neuron 1 gene (SMN1) and distal SMA (dSMA) caused by dominant mutations of the transient receptor potential vanilloid 4 gene (TRPV4). This administrative supplement to my R35 grant funded by the NINDS Landis Mentorship Award will provide funds to augment the mentorship and training of four graduate students at Johns Hopkins University School of Medicine with the long-term goal of fostering their future careers in biomedicine working to advance treatment for neurodegenerative diseases. Each student will pursue a graduate thesis project that is within the scope of research proposed in the parent R35 grant: Stephen Brown and Maddie Dent will focus on proximal SMA and Anna Bagnell and Jonathan Alevy will focus on distal SMA. They will leverage unique resources and state-of-the-art technologies to define factors limiting efficacy of current SMA therapeutics, characterize cellular and molecular mechanisms driving SMA pathology, and identify and validate novel therapeutic strategies. In order to foster their training, the first aim of the supplement is to provide regular one-on-one meetings for scientific discussion including review of relevant literature, hypothesis generation, experimental design, and data interpretation. The second aim to foster their training by enabling them to utilize cutting-edge experimental techniques to pursue their scientific hypotheses. The third aim is to support travel of students to scientific meetings for presentations and networking and the fourth aim is to create a rich community of trainees for “peer” mentoring.
期刊论文(13)
专著(0)
科研奖励(0)
会议论文
Correction to: Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism.
更正:周围神经病变:不再是治疗虚无主义的土地。
DOI: 10.1007/s13311-022-01187-x
发表时间: 2022
期刊: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
影响因子: --
作者: [Polydefkis,Michael, Sumner,CharlotteJ]
通讯作者: Sumner,CharlotteJ
DOI: 10.1038/s41591-022-01889-x
发表时间: 2022-07
期刊: NATURE MEDICINE
影响因子: 82.9
作者: [Sumner, Charlotte J., Crawford, Thomas O.]
通讯作者: Crawford, Thomas O.
DOI: 10.3233/jnd-230054
发表时间: 2023
期刊: JOURNAL OF NEUROMUSCULAR DISEASES
影响因子: 3.3
作者: [Glascock, Jacqueline, Darras, Basil T., Crawford, Thomas O., Sumner, Charlotte J., Kolb, Stephen J., DiDonato, Christine, Elsheikh, Bakri, Howell, Kelly, Farwell, Wildon, Valente, Marta, Petrillo, Marco, Tingey, Jessica, Jarecki, Jill]
通讯作者: Jarecki, Jill
DOI: 10.1002/acn3.51523
发表时间: 2022-03
期刊: Annals of clinical and translational neurology
影响因子: 5.3
作者: [Taga A, Peyton MA, Goretzki B, Gallagher TQ, Ritter A, Harper A, Crawford TO, Hellmich UA, Sumner CJ, McCray BA]
通讯作者: McCray BA
共 10 条
    Translating Pathomechanisms into Treatment for Spinal Muscular Atrophies
    • 批准号:
      10401905
    • 项目类别:
    • 资助金额:
      $100.2万
    • 财政年份:
      2021
    • 负责人:
      Charlotte Jane Sumner
    • 依托单位:
    Translating Pathomechanisms into Treatment for Spinal Muscular Atrophies
    • 批准号:
      10239537
    • 项目类别:
    • 资助金额:
      $105.88万
    • 财政年份:
      2021
    • 负责人:
      Charlotte Jane Sumner
    • 依托单位:
    Translating Pathomechanisms into Treatment for Spinal Muscular Atrophies
    • 批准号:
      10611992
    • 项目类别:
    • 资助金额:
      $100.2万
    • 财政年份:
      2021
    • 负责人:
      Charlotte Jane Sumner
    • 依托单位:
    TRPV4 links the blood-neural barrier to motor neuron dysfunction
    • 批准号:
      9916170
    • 项目类别:
    • 资助金额:
      $49.7万
    • 财政年份:
      2020
    • 负责人:
      Charlotte Jane Sumner
    • 依托单位:
    国内基金
    海外基金
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    • 批准号:
      2021JJ40433
    • 项目类别:
      省市级项目
    • 资助金额:
      --
    • 批准年份:
      2021
    • 负责人:
      孙磊
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    寄主诱导梢腐病菌AreA和CYP51基因沉默增强甘蔗抗病性机制解析
    • 批准号:
      32001603
    • 项目类别:
      青年科学基金项目
    • 资助金额:
      24.0万元
    • 批准年份:
      2020
    • 负责人:
      段真珍
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    AREA国际经济模型的移植.改进和应用
    • 批准号:
      18870435
    • 项目类别:
      面上项目
    • 资助金额:
      2.0万元
    • 批准年份:
      1988
    • 负责人:
      史树中
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