Gene therapy for haemophilia

Dr. Rob C; Hoeben

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Gene therapy for haemophilia

机译：血友病的基因治疗

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Gene therapy is an appealing prospect for the treatment of human diseases. In this chapter, I will describe the hopes that gene therapy has brought for hemophilia patients, as well as the hurdles that the researchers have encountered on the route that shall lead to the development of a clinically applicable protocol.

机译：基因疗法是治疗人类疾病的诱人前景。在本章中，我将描述基因疗法给血友病患者带来的希望，以及研究人员在研发可临床应用的治疗方案的过程中遇到的障碍。

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《Gene Therapy and Molecular Biology》 |1998年第2期|共8页
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Dr. Rob C; Hoeben;
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中图分类基础医学;
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1. The 1st WFH WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world [J] . Pierce Glenn F., Coffin Donna, Members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee, Haemophilia: the official journal of the World Federation of Hemophilia . 2019,第2期

机译：第一个WFH WFH基因治疗圆桌：了解世界各地血友病疗法的景观和挑战
2. New genes for old: Successful gene therapy for haemophilia B [J] . RobertsD.J. Transfusion medicine . 2012,第1期

机译：旧的新基因：B型血友病的成功基因治疗
3. New genes for old: Successful gene therapy for haemophilia B [J] . RobertsD.J. Transfusion medicine . 2012,第1期

机译：旧基因：血友病患者的成功基因治疗
4. National Patient Registry: A Web-based Technological Solution for Haemophilia in Portugal [C] . Leonor Teixeira, Vasco Saavedra, Beatriz Sousa Santos, International Conference on Human Systems Engineering and Design . 2019

机译：国家患者登记处：葡萄牙血友病的基于网络技术解决方案
5. Combined deficiency of factors V and VIII by chance coinheritance of parahaemophilia and haemophilia A, but not by mutations of either LMAN1 or MCFD2 [D] . 鈴木幸子 2019

机译：副血友病和A血友病的偶然一致性导致因子V和VIII的联合缺乏，但不是LMAN1或MCFD2的突变
6. Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires Argentina [O] . R. R. MONTGOMERY, P. E. MONAHAN, M. C. OZELO -1

机译：血友病A和Haemophilia的治疗基因转移的独特策略A和Haemophilia BWFH艺术术语关于治疗基因转移Buenos Aires阿根廷
7. The molecular analysis of haemophilia A: a guideline from the UK haemophilia centre doctors' organization haemophilia genetics laboratory network [O] . S. Keeney, M. Mitchell, A. Goodeve 2005

机译：血友病A的分子分析 - 普通血六级医生组织血友病遗传实验网络的指导

Gene therapy for haemophilia

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