DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS
开发针对 ALS 的 MICRORNA 靶向疗法
基本信息
- 批准号:8275481
- 负责人:
- 金额:$ 33.25万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2012
- 资助国家:美国
- 起止时间:2012-04-15 至 2017-02-28
- 项目状态:已结题
- 来源:
- 关键词:AdultAffectAmyotrophic Lateral SclerosisAnimal ModelAntisense OligonucleotidesAtrophicBinding SitesBrainCell physiologyCessation of lifeClinicCodeCoupledDataDiseaseDisease ProgressionDisease modelDoseFailureFunctional RNAGenetic TranscriptionGrantHumanImmunohistochemistryInflammationKnock-outKnockout MiceLinkMeasuresMessenger RNAMethodsMicroRNAsMicrogliaMorphologyMotor NeuronsMusNeuraxisNeurodegenerative DisordersNeurogliaOligonucleotidesPatientsPharmaceutical PreparationsPhase I Clinical TrialsPositioning AttributeProcessPublishingRespiratory FailureRespiratory MusclesRodent ModelSamplingSkeletal MuscleSpinal CordSumTechnologyTestingTherapeuticTimeToxic effectTranslatingTranslationsValidationWorkcell typechemokinecytokinedesignexperienceinhibitor/antagonistinterestmacrophagemouse modelnervous system disordernew therapeutic targetnovelnovel therapeuticstherapeutic developmenttherapeutic targettool
项目摘要
DESCRIPTION (provided by applicant): Amyotrophic lateral sclerosis is characterized by the progressive loss of motor neurons in the spinal cord, resulting in stiffness, severe weakness, atrophy of skeletal muscles, and eventual death from respiratory failure in 3-5 years. There are no current therapies that substantially slow the progression of the disease. In animal models and in samples from ALS patients, we have discovered changes in small non-coding RNA called microRNAs. We will now validate one particular microRNA as a therapeutic target and develop a method of inhibiting this microRNA using antisense oligonucleotides. We hypothesize that inhibition of this miRNA will substantially slow ALS in animal models. Given our current experience in Phase I trial using antisense oligonucleotides in ALS patients; we intend to translate our findings from this grant to a novel therapeutic for ALS.
PUBLIC HEALTH RELEVANCE: There are no medications that substantially slow the course of ALS. The results of this application have the potential to validate a novel therapeutic target as well as a method of modulating that target. The microRNA target that we have validated is likely also important for other neurodegenerative diseases besides ALS and thus the miRNA inhibitor we are developing may be widely applicable. In addition, we are pioneering the use of antisense oligonucleotide inhibitors of miRNA in the brain and spinal cord, a technology that may have therapeutic implications for many neurological disorders.
描述(由申请人提供):肌萎缩性侧索硬化症的特征是脊髓运动神经元的进行性丧失,导致僵硬、严重无力、骨骼肌萎缩,最终在3-5年内因呼吸衰竭而死亡。目前还没有能够显著减缓疾病进展的治疗方法。在动物模型和ALS患者的样本中,我们发现了小的非编码RNA(称为microRNAs)的变化。我们现在将验证一种特定的microRNA作为治疗靶点,并开发一种使用反义寡核苷酸抑制该microRNA的方法。我们假设在动物模型中,抑制这种miRNA将显著减缓ALS。鉴于我们目前在ALS患者中使用反义寡核苷酸的I期试验的经验;我们打算将我们的发现从这项资助转化为一种新的治疗ALS的方法。
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(2)
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 }}
TIMOTHY M. MILLER其他文献
TIMOTHY M. MILLER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('TIMOTHY M. MILLER', 18)}}的其他基金
Understanding SOD1 Kinetics in Amyotrophic Lateral Sclerosis
了解肌萎缩侧索硬化症中的 SOD1 动力学
- 批准号:
9282516 - 财政年份:2016
- 资助金额:
$ 33.25万 - 项目类别:
Development of an antisense oligonucleotide therapy for SOD1 Familial ALS
开发 SOD1 家族性 ALS 反义寡核苷酸疗法
- 批准号:
8724083 - 财政年份:2014
- 资助金额:
$ 33.25万 - 项目类别:
Development of an antisense oligonucleotide therapy for SOD1 Familial ALS
开发 SOD1 家族性 ALS 反义寡核苷酸疗法
- 批准号:
9110459 - 财政年份:2014
- 资助金额:
$ 33.25万 - 项目类别:
Effect of SHIFT FROM 4R TO 3R TAU on AMYLOID BETA-INDUCED COGNITIVE DEFICITS
从 4R TAU 转变为 3R TAU 对淀粉样蛋白诱发的认知缺陷的影响
- 批准号:
8492321 - 财政年份:2013
- 资助金额:
$ 33.25万 - 项目类别:
DOES A SHIFT FROM 4R TO 3R TAU PROJECT AGAINST AMYLOID BETA-INDUCED COGNITIVE DEF
从 4R 到 3R TAU 项目的转变是否可以对抗淀粉样蛋白 Beta 引起的认知缺陷
- 批准号:
8685859 - 财政年份:2013
- 资助金额:
$ 33.25万 - 项目类别:
DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS
开发针对 ALS 的 MICRORNA 靶向疗法
- 批准号:
9022530 - 财政年份:2012
- 资助金额:
$ 33.25万 - 项目类别:
DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS
开发针对 ALS 的 MICRORNA 靶向疗法
- 批准号:
8824992 - 财政年份:2012
- 资助金额:
$ 33.25万 - 项目类别:
DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS
开发针对 ALS 的 MICRORNA 靶向疗法
- 批准号:
8610957 - 财政年份:2012
- 资助金额:
$ 33.25万 - 项目类别:
DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS
开发针对 ALS 的 MICRORNA 靶向疗法
- 批准号:
8456090 - 财政年份:2012
- 资助金额:
$ 33.25万 - 项目类别:
Identifying Liver Proteins that Decrease Mutant SOD1 Misfolding and Decrease SOD1
鉴定可减少突变 SOD1 错误折叠并减少 SOD1 的肝脏蛋白
- 批准号:
8129435 - 财政年份:2010
- 资助金额:
$ 33.25万 - 项目类别:
相似海外基金
How Does Particle Material Properties Insoluble and Partially Soluble Affect Sensory Perception Of Fat based Products
不溶性和部分可溶的颗粒材料特性如何影响脂肪基产品的感官知觉
- 批准号:
BB/Z514391/1 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Training Grant
BRC-BIO: Establishing Astrangia poculata as a study system to understand how multi-partner symbiotic interactions affect pathogen response in cnidarians
BRC-BIO:建立 Astrangia poculata 作为研究系统,以了解多伙伴共生相互作用如何影响刺胞动物的病原体反应
- 批准号:
2312555 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Standard Grant
RII Track-4:NSF: From the Ground Up to the Air Above Coastal Dunes: How Groundwater and Evaporation Affect the Mechanism of Wind Erosion
RII Track-4:NSF:从地面到沿海沙丘上方的空气:地下水和蒸发如何影响风蚀机制
- 批准号:
2327346 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Standard Grant
Graduating in Austerity: Do Welfare Cuts Affect the Career Path of University Students?
紧缩毕业:福利削减会影响大学生的职业道路吗?
- 批准号:
ES/Z502595/1 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Fellowship
感性個人差指標 Affect-X の構築とビスポークAIサービスの基盤確立
建立个人敏感度指数 Affect-X 并为定制人工智能服务奠定基础
- 批准号:
23K24936 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Grant-in-Aid for Scientific Research (B)
Insecure lives and the policy disconnect: How multiple insecurities affect Levelling Up and what joined-up policy can do to help
不安全的生活和政策脱节:多种不安全因素如何影响升级以及联合政策可以提供哪些帮助
- 批准号:
ES/Z000149/1 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Research Grant
How does metal binding affect the function of proteins targeted by a devastating pathogen of cereal crops?
金属结合如何影响谷类作物毁灭性病原体靶向的蛋白质的功能?
- 批准号:
2901648 - 财政年份:2024
- 资助金额:
$ 33.25万 - 项目类别:
Studentship
Investigating how double-negative T cells affect anti-leukemic and GvHD-inducing activities of conventional T cells
研究双阴性 T 细胞如何影响传统 T 细胞的抗白血病和 GvHD 诱导活性
- 批准号:
488039 - 财政年份:2023
- 资助金额:
$ 33.25万 - 项目类别:
Operating Grants
New Tendencies of French Film Theory: Representation, Body, Affect
法国电影理论新动向:再现、身体、情感
- 批准号:
23K00129 - 财政年份:2023
- 资助金额:
$ 33.25万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
The Protruding Void: Mystical Affect in Samuel Beckett's Prose
突出的虚空:塞缪尔·贝克特散文中的神秘影响
- 批准号:
2883985 - 财政年份:2023
- 资助金额:
$ 33.25万 - 项目类别:
Studentship