Conversion of Fibroblasts to fxnal Spinal Motor Neurons Using Defined Factor
Conversion of Fibroblasts to fxnal Spinal Motor Neurons Using Defined Factor
批准号:
8664459
负责人:
Justin Kawika Ichida
金额:
$24.39万
依托单位国家:
美国
项目类别:
财政年份:
2013
资助国家:
美国
项目状态:
已结题
起止时间:
2013-01-01 至 2015-12-31
关键词:
AddressAdultAmyotrophic Lateral SclerosisCellsDNA MethylationDegenerative DisorderDisease modelDoctor of PhilosophyElectrophysiology (science)EmbryoEngineeringEpigenetic ProcessFibroblastsGene Expression ProfileGenerationsGoalsHumanIn VitroIndividualMediatingMolecularMorphologyMotor NeuronsMusNervous system structureNeurodegenerative DisordersNeuronsPatientsPhasePhenotypeProcessPropertyRegenerative MedicineResourcesRodentRouteSomatic CellSourceSpinalSpinal Muscular AtrophyStem cellsStimulusSynapsesTherapeuticTherapeutic UsesTimeabstractingcell typeembryonic stem cellin vivomotor neuron functionnerve stem celltranscription factortranslational study
中文摘要
摘要
哺乳动物的神经系统由许多不同的神经元亚型组成,每种亚型具有
其自身表型和对变性疾病的不同敏感性。虽然某些特定的神经元类型
可以从完整的啮齿动物胚胎中分离出来,也可以从干细胞中工程化,用于翻译研究,这些
这些方法是耗时的,并且许多神经元亚型是不可接近的。转录因子介导
重新编程可能为疾病建模提供更直接的神经元生成途径,
再生医学,但目前还不清楚这种方法是否可以用来创造细胞与翻译
效用在这里,我们建议确定一组转录因子足以将成纤维细胞转化为
功能性脊髓运动神经元我们将描述重编程过程,并检查分子
和功能特性,以确定它们的治疗潜力。
这些研究将提供一个患者特异性运动神经元的来源,用于研究
神经退行性疾病,证明了特定的成体细胞类型可以直接从体细胞生成。
细胞使用定义的因素,并机械解剖定义的因素重编程过程。
英文摘要
Abstract
The mammalian nervous system is composed of a multitude of distinct neuronal subtypes, each with
its own phenotype and differential sensitivity to degenerative disease. Although some specific neuronal types
can be isolated from intact rodent embryos or engineered from stem cells for translational studies, these
approaches are time-consuming and many neuronal subtypes are inaccessible. Transcription factor-mediated
reprogramming might provide a more direct route to the generation of neurons for disease modeling and
regenerative medicine, but it is currently unclear if this approach can be used to create cells with translational
utility. Here, we propose to identify a set of transcription factors sufficient to convert fibroblasts into
functional spinal motor neurons. We will characterize the reprogramming process and examine the molecular
and functional properties of the resulting motor neurons in order to determine their therapeutic potential.
These studies will provide an accessible source of patient-specific motor neurons for the study of
neurodegenerative disease, demonstrate that specific adult cell types can be directly generated from somatic
cells using defined factors, and mechanistically dissect the defined-factor reprogramming process.
期刊论文(0)
专著(0)
科研奖励(0)
会议论文
Diversity Supplement for Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD (Butler)
-
批准号:10303769
-
项目类别:
-
资助金额:$1.81万
-
财政年份:2021
-
负责人:Justin Kawika Ichida
-
依托单位:
Diversity Supplement for Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD (Santana)
-
批准号:10304057
-
项目类别:
-
资助金额:$7.92万
-
财政年份:2021
-
负责人:Justin Kawika Ichida
-
依托单位:
Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD
-
批准号:10053096
-
项目类别:
-
资助金额:$67.44万
-
财政年份:2017
-
负责人:Justin Kawika Ichida
-
依托单位:
Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD
-
批准号:10408823
-
项目类别:
-
资助金额:$62.08万
-
财政年份:2017
-
负责人:Justin Kawika Ichida
-
依托单位:
Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD
-
批准号:10647873
-
项目类别:
-
资助金额:$68.68万
-
财政年份:2017
-
负责人:Justin Kawika Ichida
-
依托单位:
Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD
-
批准号:10212468
-
项目类别:
-
资助金额:$62.32万
-
财政年份:2017
-
负责人:Justin Kawika Ichida
-
依托单位:
The Role of C9ORF72 Protein Function in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
-
批准号:9484651
-
项目类别:
-
资助金额:$1.29万
-
财政年份:2017
-
负责人:Justin Kawika Ichida
-
依托单位:
Lineage reprogramming for hearing loss: development of drug screening and gene therapy approaches
-
批准号:9292295
-
项目类别:
-
资助金额:$58.05万
-
财政年份:2016
-
负责人:Justin Kawika Ichida
-
依托单位:
Conversion of Fibroblasts to fxnal Spinal Motor Neurons Using Defined Factor
-
批准号:8605244
-
项目类别:
-
资助金额:$24.9万
-
财政年份:2013
-
负责人:Justin Kawika Ichida
-
依托单位:
Conversion of Fibroblasts to fxnal Spinal Motor Neurons Using Defined Factor
-
批准号:8804957
-
项目类别:
-
资助金额:$24.36万
-
财政年份:2013
-
负责人:Justin Kawika Ichida
-
依托单位:
Conversion of fibroblasts to functional spinal motor neurons using defined factor
-
批准号:8337701
-
项目类别:
-
资助金额:$8.76万
-
财政年份:2011
-
负责人:Justin Kawika Ichida
-
依托单位:
Conversion of fibroblasts to functional spinal motor neurons using defined factor
-
批准号:8239871
-
项目类别:
-
资助金额:$8.92万
-
财政年份:2011
-
负责人:Justin Kawika Ichida
-
依托单位:
海外基金