Liver-directed gene therapy with lentiviral vectors achieve normal levels of clotting factor VIII and IX in non-human primates

Milani M.; Canepari C.; Annoni A.; Liu T.; Plati T.; Biffi M.; Russo F.; Patarroyo-White S.; Drager D.; Ayuso E.; Peters R.; Naldini L.; Cantore A.

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Liver-directed gene therapy with lentiviral vectors achieve normal levels of clotting factor VIII and IX in non-human primates

机译：用慢病毒载体的肝脏定向基因治疗达到非人最新品中凝血因子VIII和IX的正常水平

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来源
《Human gene therapy》 |2019年第11期|共1页
作者
Milani M.; Canepari C.; Annoni A.; Liu T.; Plati T.; Biffi M.; Russo F.; Patarroyo-White S.; Drager D.; Ayuso E.; Peters R.; Naldini L.; Cantore A.;
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作者单位

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

Sanofi Paris France;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

Sanofi Paris France;

Sanofi Paris France;

Univ Nantes INSERM UMR 1089 Nantes France;

Sanofi Paris France;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

San Raffaele Telethon Inst Gene Therapy HSR TIGET Milan Italy;

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正文语种 eng
中图分类治疗学;
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1. Liver-directed gene therapy with lentiviral vectors achieve normal levels of clotting factor VIII and IX in non-human primates [J] . Milani M., Canepari C., Annoni A., Human gene therapy . 2019,第11期

机译：用慢病毒载体的肝脏定向基因治疗达到非人最新品中凝血因子VIII和IX的正常水平
2. Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates. [J] . Fischer-Lougheed JY, Tarantal AF, Shulkin I, Gene therapy . 2007,第1期

机译：使用慢病毒载体在非人类灵长类动物中抑制异种抗体产生的基因疗法。
3. The measurement of low levels of factor VIII or factor IX in hemophilia A and hemophilia B plasma by clot waveform analysis and thrombin generation assay. [J] . Matsumoto T, Shima M, Takeyama M, Journal of thrombosis and haemostasis: JTH . 2006,第2期

机译：通过血块波形分析和凝血酶生成测定法测定血友病A和血友病B血浆中低水平的VIII因子或IX因子。
4. Evaluation of Brain-Derived Neurotrophic Factor in Periodontal Tissue Regeneration: A Non-Human Primates Study [C] . Mousa R, Damien C, Janal M, Society for Biomaterials fall symposium 2012.;vol. 34. . 2012

机译：牙周组织再生中脑源性神经营养因子的评估：一项非人类灵长类动物研究
5. Gene therapy for hemophilia A: Targeting long-term hematopoietic repopulating cells with a retroviral vector expressing a bioengineered human factor VIII gene [D] . Moayeri, Morvarid 2005

机译：血友病A的基因治疗：用表达生物工程化人类因子VIII基因的逆转录病毒载体靶向长期造血的繁殖细胞
6. Variations in levels of blood clotting factors IX and X in a population of normal men: possible genetic polymorphisms. [O] . R H Lester, R C Elston, J B Graham 1972

机译：正常男性人群中凝血因子IX和X的水平变化：可能的遗传多态性。
7. 582. Expression of Therapeutic Levels of Human Clotting Factor IX in Non Human Primates from an AAV5-Based Gene Therapeutic Vector Generated Using a Fully-Scalable GMP-Compliant Production System [O] . 2012

机译：582.使用完全可扩展的GMP标准的生产系统产生的AAV5基因治疗载体中非人原始素治疗人凝血因子IX的治疗水平的表达
8. Anti-Angiogenic Gene Therapy of Prostate Cancer with Lentiviral Vectors. [R] . Yes, J. 2004

机译：抗病毒载体的抗血管生成基因治疗前列腺癌。

Liver-directed gene therapy with lentiviral vectors achieve normal levels of clotting factor VIII and IX in non-human primates

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