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Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of #946;-Thalassemia and Sickle Cell Disease

机译：改进的慢病毒载体治疗β-地中海贫血和镰状细胞病的疗效和安全性的临床前评价

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Olivier Negre; Cynthia Bartholomae; Yves Beuzard; Marina Cavazzana; Lauryn Christiansen; Celine Courne; Annette Deichmann; Maria Denaro; Edouard Dreuzy; Mitchell Finer; Raffaele Fronza; Beatrix Gillet-Legrand; Christophe Joubert; Robert Kutner; Philippe Leboulch; Leila Maouche; Anais Paulard; Francis Pierciey; Michael Rothe; Byoung Ryu; Manfred Schmidt; Christof Kalle; Emmanuel Payen; Gabor Veres;
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1. Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of beta-Thalassemia and Sickle Cell Disease [J] . Negre Olivier, Bartholomae Cynthia, Beuzard Yves, Current gene therapy . 2015,第1期

机译：改进的慢病毒载体治疗β-地中海贫血和镰状细胞病的疗效和安全性的临床前评估
2. Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (vol 21, pg 175, 2013) [J] . Scaramuzza Samantha, Biasco Luca, Ripamonti Anna, Molecular therapy: the journal of the American Society of Gene Therapy . 2016,第6期

机译：慢病毒载体转导治疗维斯科特-奥尔德里奇综合症的人CD34 +细胞的临床前安全性和功效（第21卷，第175页，2013年）
3. Preclinical Safety and Efficacy of human CD34 + Cells transduced with lentiviral vector for the treatment of wiskott-aldrich syndrome [J] . ScaramuzzaS., BiascoL., RipamontiA., Molecular therapy: the journal of the American Society of Gene Therapy . 2013,第1期

机译：慢病毒载体转导的人CD34 +细胞治疗wiskott-aldrich综合征的临床前安全性和功效
4. Evaluation of the efficacy and safety of Nuflor injectable solution (15 mg/kg twice 48 hours apart) in the treatment of swine respiratory disease (SRD) [C] . Robert Zolynas, Jean Cao, Robert Simmons Annual Meeting of the American Association of Swine Veterinarians . 2003

机译：评价Nuflor可注射溶液（15mg / kg的48小时48小时）治疗猪呼吸道疾病（SRD）的疗效和安全性
5. Correction of mouse models of sickle cell disease and beta-thalassemia using stem cell and genetic-based therapy. [D] . Levasseur, Dana N. 2003

机译：使用干细胞和基于基因的疗法校正镰状细胞病和β地中海贫血的小鼠模型。
6. Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease [O] . Olivier Negre, Cynthia Bartholomae, Yves Beuzard, -1

机译：改进的慢病毒载体治疗β-地中海贫血和镰状细胞病的疗效和安全性的临床前评估
7. Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome [O] . Scaramuzza, S, Biasco, L, Ripamonti, A, 2013

机译：慢病毒载体转导的人CD34（+）细胞治疗Wiskott-Aldrich综合征的临床前安全性和功效
8. Sickle Cell Disease: Screening, Diagnosis, Management, and Counseling in Newborns and Infants. Clinical Practice Guideline Number 6; Sickle Cell Disease: Comprehensive Screening and Management in Newborns and Infants. Quick Reference Guide for Clinicians Number 6; Sickle Cell Disease in Newborns and Infants. A Guide for Parents [R] . 1993

机译：镰状细胞病：新生儿和婴儿的筛查，诊断，管理和咨询。临床实践指南第6号;镰状细胞病：新生儿和婴儿的综合筛查和管理。第6号临床医生快速参考指南;新生儿和婴儿的镰状细胞病。家长指南

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of #946;-Thalassemia and Sickle Cell Disease

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