GENE DELIVERY TO THE GROWING MURINE LIVER USING A HYBRID RAAV/PIGGYBAC SYSTEM RESULTS IN STABLE CORRECTION OF SEVERE METABOLIC LIVER DISEASE PHENOTYPES

Cunningham Sharon C.; Siew Susan M.; Hallwirth Claus V.; Bolitho Christine; Sasaki Natsuki; Garg Gagan; Michael Iacovos P.; Hetherington Nicola A.; Carpenter Kevin; de Alencastro Gustavo; Nagy Andras; Alexander Ian E.

首页> 外文期刊>The journal of gene medicine >GENE DELIVERY TO THE GROWING MURINE LIVER USING A HYBRID RAAV/PIGGYBAC SYSTEM RESULTS IN STABLE CORRECTION OF SEVERE METABOLIC LIVER DISEASE PHENOTYPES

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GENE DELIVERY TO THE GROWING MURINE LIVER USING A HYBRID RAAV/PIGGYBAC SYSTEM RESULTS IN STABLE CORRECTION OF SEVERE METABOLIC LIVER DISEASE PHENOTYPES

机译：使用杂交RAAV /猪皮草系统将基因传递到生长的尿液中，可稳定纠正严重的代谢性肝病表型

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来源
《The journal of gene medicine》 |2015年第9期|共2页
作者
Cunningham Sharon C.; Siew Susan M.; Hallwirth Claus V.; Bolitho Christine; Sasaki Natsuki; Garg Gagan; Michael Iacovos P.; Hetherington Nicola A.; Carpenter Kevin; de Alencastro Gustavo; Nagy Andras; Alexander Ian E.;
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正文语种 eng
中图分类医学遗传学;
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1. GENE DELIVERY TO THE GROWING MURINE LIVER USING A HYBRID RAAV/PIGGYBAC SYSTEM RESULTS IN STABLE CORRECTION OF SEVERE METABOLIC LIVER DISEASE PHENOTYPES [J] . Cunningham Sharon C., Siew Susan M., Hallwirth Claus V., The journal of gene medicine . 2015,第8a9期

机译：使用杂交RAAV /猪皮草系统将基因传递到生长的尿液中，可稳定纠正严重的代谢性肝病表型
2. COMPARISON OF HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS/TRANSPOSON GENE DELIVERY SYSTEMS BASED ON PIGGYBAC AND SLEEPING BEAUTY IN THE LIVER [J] . Cunningham Sharon C., Sasaki Natsuki, Kok Cindy, The journal of gene medicine . 2018,第1期

机译：基于Piggybac和肝脏睡眠美女的杂种重组腺相关病毒/转座子基因递送系统的比较
3. Long-Term Disease Correction and Reduced Liver Tumorigenesis Using Hybrid Recombinant AAV-Piggybac Transposon Mediated Gene Therapy in the Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3) Mouse Model in vivo [J] . Siew Susan M., Zhu Erhua, Tay Szun, Hepatology: Official Journal of the American Association for the Study of Liver Diseases . 2017,第Suppla1期

机译：使用杂种重组AAV-PICGGYBAC转座子介导的基因治疗在体内逐步家族性肝内胆碱型3（PFIC3）小鼠模型中使用杂种重组AAV-PICGGY-PIGGYAS转座子介导的基因治疗的长期疾病矫正
4. Macrophage-Targeted Alginate Nanoparticles as a Non-Condensing Murine IL-10 Gene Delivery System for the Treatment of Experimental Arthritis [C] . Shardool Jain, Mansoor Amiji Society for Biomaterials annual meeting and exposition . 2013

机译：巨噬细胞靶向藻酸盐纳米颗粒作为非凝结小鼠IL-10基因递送系统，用于治疗实验性关节炎
5. Properties and techniques of intravascular rAAV6 gene delivery to striated muscle. [D] . Schultz, Brian Richard. 2009

机译：血管内rAAV6基因传递至横纹肌的特性和技术。
6. Systemic delivery of rAAV6-microdystrophin preserves muscle function and extends lifespan in a murine model of severe muscular dystrophy [O] . Paul Gregorevic, James M. Allen, Elina Minami, -1

机译：在严重肌肉营养不良的小鼠模型中rAAV6-微肌营养不良蛋白的全身递送可保留肌肉功能并延长寿命
7. Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBac transposase gene delivery system [O] . Cunningham, Sharon C, Siew, Susan M, Nagy, Andras, 2015

机译：使用重组重组腺相关病毒/ piggyBac转座酶基因传递系统对正在生长的鼠肝中严重尿素循环缺陷的校正模型
8. Development of a Novel Targeted RNAi Delivery Technology inTherapies for Metabolic Diseases. [R] . Czech, M. P. 2017

机译：新型靶向RNai递送技术在代谢疾病治疗中的应用。

GENE DELIVERY TO THE GROWING MURINE LIVER USING A HYBRID RAAV/PIGGYBAC SYSTEM RESULTS IN STABLE CORRECTION OF SEVERE METABOLIC LIVER DISEASE PHENOTYPES

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