Frataxin Restoration in the Nervous System: Possibilities for Gene Therapy.
作者信息
Lynch David R, Kichula Elizabeth, Lin Hong
机构信息
Division of Neurology, Children's Hospital of Philadelphia, 502 Abramson Research Center, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Division of Neurology, Children's Hospital of Philadelphia, 502 Abramson Research Center, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
出版信息
Mol Ther. 2018 Aug 1;26(8):1880-1882. doi: 10.1016/j.ymthe.2018.06.006. Epub 2018 Jun 30.
Abstract
摘要
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本文引用的文献
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Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia.新型弗里德里希共济失调模型中基因治疗快速完全逆转感觉共济失调。
Mol Ther. 2018 Aug 1;26(8):1940-1952. doi: 10.1016/j.ymthe.2018.05.006. Epub 2018 May 28.
2
Early cerebellar deficits in mitochondrial biogenesis and respiratory chain complexes in the KIKO mouse model of Friedreich ataxia.弗里德里希共济失调症 KIKO 小鼠模型中线粒体生物发生和呼吸链复合物的早期小脑缺陷。
Dis Model Mech. 2017 Nov 1;10(11):1343-1352. doi: 10.1242/dmm.030502.
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Purkinje cell injury, structural plasticity and fusion in patients with Friedreich's ataxia.弗里德里希共济失调患者浦肯野细胞损伤、结构可塑性和融合。
Acta Neuropathol Commun. 2016 May 23;4(1):53. doi: 10.1186/s40478-016-0326-3.
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Longitudinal magnetic resonance imaging study shows progressive pyramidal and callosal damage in Friedreich's ataxia.磁共振成像纵向研究显示弗里德里希共济失调中锥体束和胼胝体的进行性损伤。
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