Mammalian motor neuron SMN screens

哺乳动物运动神经元 SMN 筛查

基本信息

  • 批准号:
    8375844
  • 负责人:
  • 金额:
    $ 30.38万
  • 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:
  • 项目状态:
    未结题

项目摘要

Spinal muscular atrophy (SMA) is a common childhood autosomal recessive disease caused by mutafions in the Survival of Motor Neuron 1 (SMN1) gene. One of the primary features of SMA is the progressive loss of neuromuscular function that is often fatal, making SMA the leading genetic cause of death in infants and young children. Motor neuron death is a significant feature of this disease, but some recent informafion suggests that muscle dysfuncfion or malformafion may also occur. While SMN appears to have mulfiple cellular roles, and it is not yet clear which of them support neuromuscular development and health, a reasonable amount of patient informafion indicates that higher levels of SMN expression are associated with less severe cases of disease. This suggests a clear therapeutic strategy: namely, identifying the pathways and, ultimately, drug classes that increase SMN levels. However, there are alternate strategies, one of which is finding pathways that function independently of SMN and are corrective when SMN levels are reduced. To accomplish this, we and our collaborators have carried out two sets of screens. The first set used chemical and biological libraries to search for compounds that increase amounts of SMN in mouse motor neurons and other cells. The second set used genetic methods to find genes that can ameliorate SMA phenotypes in fiy and worm models. We will establish a set of key phenotypic assays to test all of the compounds and genes that come out of the screens. These will include mouse motor neuron survival, skeletal muscle development and neuromuscular junction formafion. In addition, we will test these compounds and genes on human motor neurons produced from induced pluripotent stem (iPS) cells made from an SMA patient. Targets identified from chemical screens will be cross-validated in genetic models. Thus, hits from all the screens will be evaluated and compared rigorously. Finally, compounds indentified from these screens will be tested in mouse SMA models. The end result of this work should be thoroughly characterized compounds that can potenfially be used to develop therapeufics for this childhood disease.
脊髓性肌萎缩症(SMA)是一种常见的儿童常染色体隐性遗传病

项目成果

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Lee L Rubin其他文献

Lee L Rubin的其他文献

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{{ truncateString('Lee L Rubin', 18)}}的其他基金

Direct and Indirect Effects of GDF11 in the Aging Central Nervous System
GDF11 对衰老中枢神经系统的直接和间接影响
  • 批准号:
    10661530
  • 财政年份:
    2020
  • 资助金额:
    $ 30.38万
  • 项目类别:
Direct and Indirect Effects of GDF11 in the Aging Central Nervous System
GDF11 对衰老中枢神经系统的直接和间接影响
  • 批准号:
    10263387
  • 财政年份:
    2020
  • 资助金额:
    $ 30.38万
  • 项目类别:
Direct and Indirect Effects of GDF11 in the Aging Central Nervous System
GDF11 对衰老中枢神经系统的直接和间接影响
  • 批准号:
    10206805
  • 财政年份:
    2020
  • 资助金额:
    $ 30.38万
  • 项目类别:
Direct and Indirect Effects of GDF11 in the Aging Central Nervous System
GDF11 对衰老中枢神经系统的直接和间接影响
  • 批准号:
    10437025
  • 财政年份:
    2020
  • 资助金额:
    $ 30.38万
  • 项目类别:
Identifying and Correcting Dementia-Associated Changes in the Blood-Brain Barrier
识别和纠正与痴呆相关的血脑屏障变化
  • 批准号:
    10031380
  • 财政年份:
    2020
  • 资助金额:
    $ 30.38万
  • 项目类别:
Mammalian motor neuron SMN screens
哺乳动物运动神经元 SMN 筛查
  • 批准号:
    8509038
  • 财政年份:
  • 资助金额:
    $ 30.38万
  • 项目类别:
Mammalian motor neuron SMN screens
哺乳动物运动神经元 SMN 筛查
  • 批准号:
    8013214
  • 财政年份:
  • 资助金额:
    $ 30.38万
  • 项目类别:
Mammalian motor neuron SMN screens
哺乳动物运动神经元 SMN 筛查
  • 批准号:
    8704296
  • 财政年份:
  • 资助金额:
    $ 30.38万
  • 项目类别:
Mammalian motor neuron SMN screens
哺乳动物运动神经元 SMN 筛查
  • 批准号:
    8291241
  • 财政年份:
  • 资助金额:
    $ 30.38万
  • 项目类别:

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