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A novel BAFopathy syndrome: Functional genetics using human stem cells and gene editing technologies

A novel BAFopathy syndrome: Functional genetics using human stem cells and gene editing technologies
一种新型 BAF 病综合征：利用人类干细胞和基因编辑技术进行功能遗传学
批准号：
357724
负责人：
Ernst Carl P
金额：
$67.43万
依托单位：
CIUSSS de l'Ouest-de-l'Ile-de-Montral-Douglas Hospital
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2016
资助国家：
加拿大
项目状态：
已结题
起止时间：
2016-10-01 至 2021-10-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=357724&lang=en
关键词：
Actl6b Genetics Neurodevelopment Neuroscience

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中文摘要
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英文摘要
Certain molecular complexes are required for brain cells to mature properly. In mammals including humans, a complex called BAF is known to be essential for brain development. BAF is made up of several subunits, some of which are already associated with
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会议论文
Developing Gene Therapies for Rare Brain Disorders
Mechanisms of FOXG1 deficiency in brain cell proliferation and patterning
Development and preclinical testing in human cell models and transgenic mice of a novel treatment for Schinzel-Giedion Syndrome
Canada Research Chair - Tier 2
  • 批准号：
    378537
  • 项目类别：
  • 资助金额：
    $36.43万
  • 财政年份：
    2017
  • 负责人：
    Ernst Carl P
  • 依托单位：
    McGill University
国内基金
海外基金
Journal of Genetics and Genomics