Preclinical research in Rett syndrome: setting the foundation for translational success.
Preclinical research in Rett syndrome: setting the foundation for translational success.
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DOI：
10.1242/dmm.011007
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发表时间：
2012-11
影响因子：
4.3
通讯作者：
Mamounas LA
中科院分区：
文献类型：
--
作者：
Katz DM;Berger-Sweeney JE;Eubanks JH;Justice MJ;Neul JL;Pozzo-Miller L;Blue ME;Christian D;Crawley JN;Giustetto M;Guy J;Howell CJ;Kron M;Nelson SB;Samaco RC;Schaevitz LR;St Hillaire-Clarke C;Young JL;Zoghbi HY;Mamounas LA
2011年9月，美国国家神经疾病和中风研究所（NINDS）、尤尼斯·肯尼迪·施莱佛国家儿童健康和人类发展研究所（NICHD）、国际雷特综合征基金会（IRSF）和雷特综合征研究信托基金会（RSRT）召开了一次研讨会，与会者包括来自美国国立卫生研究院（NIH）的基础科学家、临床医生和代表，美国食品和药物管理局（FDA），制药行业和私人基金会评估Rett综合征（RTT）动物研究的最新技术水平。研讨会的目的是确定关键的知识差距，并建议在潜在的新RTT疗法的临床前评价中使用动物模型的科学优先事项和最佳实践。本综述总结了研讨会的结果和与会者广泛的后续讨论，包括：（1）全面总结了迄今为止RTT小鼠模型的生理和行为表型，以及需要进一步表型分析以提高这些模型在转化研究中的实用性的领域;（2）讨论小鼠模型之间的遗传差异和实验室之间的方法学差异分别对表型性状的表达和分析的影响;以及（3）RTT研究人员社区可以实施的标准定义，以进行严格的临床前研究设计和透明的报告，以确保启动昂贵的临床试验的决策基于可靠的临床前数据。
In September of 2011, the National Institute of Neurological Disorders and Stroke (NINDS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the International Rett Syndrome Foundation (IRSF) and the Rett Syndrome Research Trust (RSRT) convened a workshop involving a broad cross-section of basic scientists, clinicians and representatives from the National Institutes of Health (NIH), the US Food and Drug Administration (FDA), the pharmaceutical industry and private foundations to assess the state of the art in animal studies of Rett syndrome (RTT). The aim of the workshop was to identify crucial knowledge gaps and to suggest scientific priorities and best practices for the use of animal models in preclinical evaluation of potential new RTT therapeutics. This review summarizes outcomes from the workshop and extensive follow-up discussions among participants, and includes: (1) a comprehensive summary of the physiological and behavioral phenotypes of RTT mouse models to date, and areas in which further phenotypic analyses are required to enhance the utility of these models for translational studies; (2) discussion of the impact of genetic differences among mouse models, and methodological differences among laboratories, on the expression and analysis, respectively, of phenotypic traits; and (3) definitions of the standards that the community of RTT researchers can implement for rigorous preclinical study design and transparent reporting to ensure that decisions to initiate costly clinical trials are grounded in reliable preclinical data.
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影响因子：
6.1
作者：
Asaka, Y;Jugloff, DGM;Fitzsimonds, RM
通讯作者：
Fitzsimonds, RM
影响因子：
16.2
作者：
Chang, QA;Khare, G;Jaenisch, R
通讯作者：
Jaenisch, R
影响因子：
1.7
作者：
BELICHENKO, PV;OLDFORS, A;DAHLSTROM, A
通讯作者：
DAHLSTROM, A
影响因子：
6.1
作者：
Chapleau CA;Calfa GD;Lane MC;Albertson AJ;Larimore JL;Kudo S;Armstrong DL;Percy AK;Pozzo-Miller L
通讯作者：
Pozzo-Miller L
影响因子：
3.7
作者：
Boggio EM;Lonetti G;Pizzorusso T;Giustetto M
通讯作者：
Giustetto M