LONG-TERM REPEAT-DOSE PHARMACOLOGY STUDIES WITH HUMAN MUT MRNA DEMONSTRATE DURABLE FUNCTIONAL BENEFIT IN TWO MOUSE MODELS OF METHYLMALONIC ACIDEMIA (MMA)

An Ding; Frassetto Andrea; Jacquinet Eric; Eybye Marianne; Milano Joe; DeAntonis Christie; Vi Nguyen; Winkler Felicity; Laureano Rodrigo; Pierson Nancy; Milton Jaclyn; Sabnis Stacy; Elkus Maxwell; Smith Michael; Lukacs Christine; Parsons Don; Guey Lin

首页> 外文期刊>Molecular genetics and metabolism >LONG-TERM REPEAT-DOSE PHARMACOLOGY STUDIES WITH HUMAN MUT MRNA DEMONSTRATE DURABLE FUNCTIONAL BENEFIT IN TWO MOUSE MODELS OF METHYLMALONIC ACIDEMIA (MMA)

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LONG-TERM REPEAT-DOSE PHARMACOLOGY STUDIES WITH HUMAN MUT MRNA DEMONSTRATE DURABLE FUNCTIONAL BENEFIT IN TWO MOUSE MODELS OF METHYLMALONIC ACIDEMIA (MMA)

机译：具有人mutmRNA的长期重复剂量药理学研究表明，两种小鼠甲基醛酸血症（MMA）的耐用功能益处

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来源
《Molecular genetics and metabolism》 |2019年第3期|共2页
作者
An Ding; Frassetto Andrea; Jacquinet Eric; Eybye Marianne; Milano Joe; DeAntonis Christie; Vi Nguyen; Winkler Felicity; Laureano Rodrigo; Pierson Nancy; Milton Jaclyn; Sabnis Stacy; Elkus Maxwell; Smith Michael; Lukacs Christine; Parsons Don; Guey Lin;
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Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

Moderna Inc Cambridge MA USA;

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原文格式 PDF
正文语种 eng
中图分类内分泌腺疾病及代谢病;
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1. LONG-TERM REPEAT-DOSE PHARMACOLOGY STUDIES WITH HUMAN MUT MRNA DEMONSTRATE DURABLE FUNCTIONAL BENEFIT IN TWO MOUSE MODELS OF METHYLMALONIC ACIDEMIA (MMA) [J] . An Ding, Frassetto Andrea, Jacquinet Eric, Molecular genetics and metabolism . 2019,第3期

机译：具有人mutmRNA的长期重复剂量药理学研究表明，两种小鼠甲基醛酸血症（MMA）的耐用功能益处
2. BEHAVIORAL PHENOTYPE IN A LIVER TRANSGENIC MOUSE MODEL (MUT-/-;TgINS-ALB-MUT) OF METHYLMALONIC ACIDEMIA [J] . Fraser Jamie, Arnold Madeline, Gagne Jean, Molecular genetics and metabolism . 2016,第3期

机译：甲基丙二酸肝转基因小鼠模型（MUT-/-; Tgins-ALB-MUT）的行为表型
3. Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial [J] . Marwan Nashabat, Abdulrahman Obaid, Fuad Al Mutairi, BMC Pediatrics . 2019,第1期

机译：评估丙醇酸血症（PA）或甲基醛酸血症患者使用陨石酸的长期有效性：用于随机对照试验的研究方案
4. Repopulation of diseased mouse liver with rodent and human adult primary hepatocytes and long-term survival of transplanted hepatocytes in the uPA/RAG-2 mouse model [C] . M. DANDRI, M. R. BURDA, J. PETERSEN, International Congress on Thrombosis . 2002

机译：用啮齿动物和人成人原发性肝细胞的患病小鼠肝脏的重新迁移，upA / Rag-2小鼠模型中移植肝细胞的长期存活
5. Pharmacological and functional characterization of the sigma-1 receptor: Studies utilizing sigma-1 receptor photoprobes and the sigma-1 null homozygous mouse. [D] . Fontanilla, Dominique A. 2010

机译：sigma-1受体的药理和功能表征：利用sigma-1受体光电探针和sigma-1 null纯合小鼠的研究。
6. Systematic literature review and meta-analysis on the epidemiology of methylmalonic acidemia (MMA) with a focus on MMA caused by methylmalonyl-CoA mutase (mut) deficiency [O] . Tímea Almási, Lin T. Guey, Christine Lukacs, 2019

机译：关于甲基丙二酸血症（MMA）流行病学的系统文献综述和荟萃分析重点是由甲基丙二酸-CoA突变酶（mut）缺乏引起的MMA
7. Systematic literature review and meta-analysis on the epidemiology of methylmalonic acidemia (MMA) with a focus on MMA caused by methylmalonyl-CoA mutase (mut) deficiency [O] . Tímea Almási, Lin T. Guey, Christine Lukacs, 2019

机译：对甲基丙酮醛酸血症（MMA）流行病学的系统文献综述及荟萃分析，重点是由甲基甘油基 - 辅酶（MUT）缺乏引起的MMA

LONG-TERM REPEAT-DOSE PHARMACOLOGY STUDIES WITH HUMAN MUT MRNA DEMONSTRATE DURABLE FUNCTIONAL BENEFIT IN TWO MOUSE MODELS OF METHYLMALONIC ACIDEMIA (MMA)

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