Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.-论文-万方医学网

第一作者： Maria,Garcia-Gomez

第一单位： Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) - Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Madrid, Spain.;Advanced Therapies Mixed Unit. Instituto de Investigación Sanitaria-Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain.

作者： Maria,Garcia-Gomez ^[1] ; Andrea,Calabria ^[2] ; Maria,Garcia-Bravo ^[1] ; Fabrizio,Benedicenti ^[2] ; Penelope,Kosinski ^[3] ; Sergio,López-Manzaneda ^[1] ; Collin,Hill ^[3] ; María,Del Mar Mañu-Pereira ^[4] ; Miguel A,Martín ^[1] ; Israel,Orman ^[1] ; Joan-LLuis,Vives-Corrons ^[4] ; Charles,Kung ^[3] ; Axel,Schambach ^[5] ; Shengfang,Jin ^[3] ; Juan A,Bueren ^[1] ; Eugenio,Montini ^[2] ; Susana,Navarro ^[1] ; Jose C,Segovia ^[1]

作者单位： Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) - Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Madrid, Spain.;Advanced Therapies Mixed Unit. Instituto de Investigación Sanitaria-Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain. ^[1] San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy. ^[2] Agios Pharmaceuticals, Cambridge, MA, USA. ^[3] Red Cell Pathology Laboratory. Hospital Clínic of Barcelona - Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain. ^[4] Institute of Experimental Hematology at Hannover Medical Hospital, Hannover, Germany. ^[5]

医学主题词贫血, 溶血性, 先天性非球形红细胞性(Anemia, Hemolytic, Congenital Nonspherocytic);动物(Animals);血细胞(Blood Cells);细胞分化(Cell Differentiation);疾病模型, 动物(Disease Models, Animal);红细胞(Erythrocytes);红细胞生成(Erythropoiesis);遗传载体(Genetic Vectors);糖酵解(Glycolysis);造血干细胞移植(Hematopoietic Stem Cell Transplantation);造血干细胞(Hematopoietic Stem Cells);人类(Humans);慢病毒属(Lentivirus);代谢网络和途径(Metabolic Networks and Pathways);代谢组(Metabolome);代谢组学(Metabolomics);小鼠(Mice);小鼠, 转基因(Mice, Transgenic);突变(Mutation);表型(Phenotype);丙酮酸激酶(Pyruvate Kinase);丙酮酸代谢障碍, 先天性(Pyruvate Metabolism, Inborn Errors);转导, 遗传(Transduction, Genetic)

DOI 10.1038/mt.2016.87

PMID 27138040

发布时间 2018-11-13

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Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.

Molecular therapy

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Molecular therapy

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Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.

Molecular therapy

相似文献

同项目论文

Molecular therapy

相关期刊

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