Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis

Rozenberg Alejandra J.; Lykken Erik; Spratt Kaye; Miller Timothy J.; Gray Steven J.

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Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis

机译：CLN1基因治疗的组合给药在婴儿神经元曲线唇裂病毒态病变的小鼠模型中延伸了寿命

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来源
《Molecular genetics and metabolism》 |2018年第2期|共1页
作者
Rozenberg Alejandra J.; Lykken Erik; Spratt Kaye; Miller Timothy J.; Gray Steven J.;
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作者单位

Univ N Carolina Chapel Hill NC USA;

Univ N Carolina Chapel Hill NC USA;

Abeona Therapeut Inc Cleveland OH USA;

Abeona Therapeut Inc Cleveland OH USA;

Univ N Carolina Chapel Hill NC 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. Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis [J] . Rozenberg Alejandra J., Lykken Erik, Spratt Kaye, Molecular genetics and metabolism . 2018,第2期

机译：CLN1基因治疗的组合给药在婴儿神经元曲线唇裂病毒态病变的小鼠模型中延伸了寿命
2. Intrathecal and intravenous combination gene therapy in the mouse model of infantile neuronal ceroid lipofuscinosis extends lifespan and improves behavioral outcomes in moderately affected mice [J] . Gray Steven J., Rozenberg Alejandra, Lykken Erik, Molecular genetics and metabolism . 2019,第2期

机译：在婴儿神经元曲线型唇裂病变的小鼠模型中的鞘内和静脉内联合基因治疗延伸了寿命并改善了中等受影响的小鼠的行为结果
3. Early intrathecal gene therapy extends lifespan and improves quality of life in a mouse model for infantile neuronal ceroid lipofuscinosis [J] . Rozenberg Alejandra J., Gray Steven J. Molecular genetics and metabolism . 2017,第1a2期

机译：早期的鞘内基因治疗延长寿命并提高小鼠模型中的生命质量，用于婴儿神经元曲线型唇脓性
4. Calpain proteolytic systems in neurodegeneration in brains of South Hampshire sheep with neuronal ceroid lipofuscinosis [C] . Hannah Y.Y. Lee, Graham Kay, James D. Morton Incorporating of the Australian Society for Biochemistry and Molecular Biology Combio . 2009

机译：南汉普郡毛毛母细胞蛋白质蛋白水解系统，具有神经元曲线唇脓性
5. Gene therapy and enzyme replacement in a mouse model of late infantile neuronal ceroid lipofuscinosis. [D] . Chang, Michael Chia Hsiu. 2009

机译：晚期婴儿神经元类固醇脂褐藻病小鼠模型中的基因治疗和酶替代。
6. Pathogenesis and Therapies for Infantile Neuronal Ceroid Lipofuscinosis (infantile CLN1 disease) [O] . Jacqueline A. Hawkins-Salsbury, Jonathan D. Cooper, Mark S. Sands -1

机译：婴儿神经元性脂质脂肪增多症（婴儿CLN1病）的发病机制和治疗方法
7. Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease) [O] . Hawkins-Salsbury, Jacqueline A., Cooper, Jonathan D., Sands, Mark S. 2013

机译：婴幼儿神经元类脂褐藻病（婴儿CLN1疾病）的发病机制和治疗方法

Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis

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