Dark-adaptation functions in molecularly confirmed achromatopsia and the implications for assessment in retinal therapy trials

AboshihaJ.; LuongV.; CowingJ.; DubisA.M.; BainbridgeJ.W.; AliR.R.; WebsterA.R.; MooreA.T.; FitzkeF.W.; MichaelidesM.

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Dark-adaptation functions in molecularly confirmed achromatopsia and the implications for assessment in retinal therapy trials

机译：分子确诊的色盲症中的暗适应功能及其对视网膜治疗试验评估的意义

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PURPOSE: To describe the dark-adaptation (DA) functions in subjects with molecularly proven achromatopsia (ACHM) using refined testing conditions with a view to guiding assessment in forthcoming gene therapy trials.

机译：目的：使用改良的测试条件描述分子证明无色症（ACHM）受试者的暗适应（DA）功能，以指导即将进行的基因治疗试验中的评估。

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《Investigative ophthalmology & visual science》 |2014年第10期|共10页
作者
AboshihaJ.; LuongV.; CowingJ.; DubisA.M.; BainbridgeJ.W.; AliR.R.; WebsterA.R.; MooreA.T.; FitzkeF.W.; MichaelidesM.;
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正文语种 eng
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关键词
Achromatopsia; Dark adaptation; Gene therapy; Rod monochromatism; Rod vision;

机译：色盲;黑暗适应;基因治疗;杆单色症;杆视觉;

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1. Dark-adaptation functions in molecularly confirmed achromatopsia and the implications for assessment in retinal therapy trials [J] . AboshihaJ., LuongV., CowingJ., Investigative ophthalmology & visual science . 2014,第10期

机译：分子确诊的色盲症中的暗适应功能及其对视网膜治疗试验评估的意义
2. Retinal morphology of patients with achromatopsia during early childhood: Implications for gene therapy [J] . YangP., MichaelsK.V., CourtneyR.J., JAMA ophthalmology . 2014,第7期

机译：儿童期色盲症患者的视网膜形态：基因治疗的意义
3. Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia [J] . Ofri Ron, Averbukh Edward, Ezra-Elia Raaya, Human gene therapy . 2018,第12期

机译：六年并计数：基因增强治疗CNGA3阿基罗麦芽糖肽的绵羊模型后，恢复光学视网膜功能和视觉行为
4. Molecular Dissemination into Local and Systemic Tissues is Regulated by Molecular Size and Tumor Vascular Status: Implications for Targeted Drug Delivery in Cancer Immunotherapy [C] . Nathan A. Rohner, Susan N. Thomas Society for Biomaterials annual meeting and exposition . 2015

机译：分子散布到局部和全身组织中受分子大小和肿瘤血管状态的调节：在癌症免疫疗法中靶向药物递送的意义。
5. Functional investigation of the EWS /ATF1 fusion protein: Implications for molecular therapy in clear cell sarcoma. [D] . Olsen, Randall James. 2001

机译：EWS / ATF1融合蛋白的功能研究：对透明细胞肉瘤的分子治疗的意义。
6. Dark-Adaptation Functions in Molecularly Confirmed Achromatopsia and the Implications for Assessment in Retinal Therapy Trials [O] . Jonathan Aboshiha, Vy Luong, Jill Cowing, -1

机译：分子确证的色盲症中的暗适应功能及其在视网膜治疗试验中评估的意义
7. Retinal structure and function in achromatopsia : implications for gene therapy. [O] . Sundaram V., Wilde C., Aboshiha J., 2014

机译：色盲症的视网膜结构和功能：对基因治疗的影响。

Dark-adaptation functions in molecularly confirmed achromatopsia and the implications for assessment in retinal therapy trials

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