Dose-ranging comparison of choroid plexus-directed versus pan-neuronal-directed recombinant AAV gene therapy in a murine model of alpha-mannosidosis

Choi Eun-Young; Kaler Stephen G.

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Dose-ranging comparison of choroid plexus-directed versus pan-neuronal-directed recombinant AAV gene therapy in a murine model of alpha-mannosidosis

机译：剂量范围比较脉络丛导向与泛神经元定向的重组AAV基因治疗α-甘露糖病的鼠模型

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《Molecular genetics and metabolism》 |2017年第2期|共1页
作者
Choi Eun-Young; Kaler Stephen G.;
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NIH Bldg 10 Bethesda MD 20892 USA;

NIH Bldg 10 Bethesda MD 20892 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. Dose-ranging comparison of choroid plexus-directed versus pan-neuronal-directed recombinant AAV gene therapy in a murine model of alpha-mannosidosis [J] . Choi Eun-Young, Kaler Stephen G. Molecular genetics and metabolism . 2017,第1a2期

机译：剂量范围比较脉络丛导向与泛神经元定向的重组AAV基因治疗α-甘露糖病的鼠模型
2. Choroid plexus-directed viral gene therapy for alpha-mannosidosis, a prototypical lysosomal disease [J] . Choi Eun-Young, Baker Eva, Kaler Stephen G. Molecular genetics and metabolism . 2015,第2期

机译：脉络丛定向病毒基因治疗α-甘露聚糖病，一种典型的溶酶体病
3. AAAV5-mediated gene therapy with choroid plexus-directed alpha-n-acetyl-glucosaminidase expression in Sanfilippo syndrome type B mice [J] . Kan Shih-hsin, Le Steven Q., Dickson Patricia I. Molecular genetics and metabolism . 2017,第1a2期

机译：AAAV5介导的基因治疗，具有脉络丛导向的α-N-乙酰氨基酚胺酶表达在Sanfilippo综合征B小鼠中
4. Painless versus conventional photodynamic therapy for treatment of actinic keratosis: Comparison of cell death and immune response in a murine model [C] . Sanjay Anand, Anton Yasinchak, Mukul Govande, Optical Methods for Tumor Treatment and Detection: Mechanisms and Techniques in Photodynamic Therapy XXVIII . 2019

机译：无痛与传统光动力疗法治疗光化性角化病的比较：鼠模型中细胞死亡和免疫反应的比较
5. Recombinant AAV gene therapy and delivery for Alzheimer's disease. [D] . Carty, Nikisha Christine. 2009

机译：重组AAV基因治疗和阿尔茨海默氏病的递送。
6. Complete correction of hyperphenylalaninemia following liver-directed recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria [O] . CO Harding, MB Gillingham, K Hamman, -1

机译：肝定向重组AAV2 / 8载体介导的基因治疗小鼠苯丙酮尿症后完全纠正高苯丙氨酸血症
7. 548. Recombinant AAV3 Serotype Vectors Generated by Using AAV3 ITRs and AAV3 Rep Proteins Efficiently Transduce Human Liver Cancer Cell Lines In Vitro and Human Liver Tumors in a Murine Xenograft Model In Vivo [O] . Pediatrics, University of Florida College of Medicine, Gainesville, FL ( host institution ), Ling, Chen ( UF author ), Yin, Zifei ( author ), 2016

机译：548.通过使用AAV3 ITR和AAV3 Rep蛋白产生的重组AAV3血清型载体在小鼠异种移植模型体内有效地体外转导人肝癌细胞系和人肝肿瘤。

Dose-ranging comparison of choroid plexus-directed versus pan-neuronal-directed recombinant AAV gene therapy in a murine model of alpha-mannosidosis

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