Preclinical biosafety studies of lentiviral vector-mediated gene therapy in erythrocyte pyruvate kinase deficiency

Quintana-Bustamante O.; Sanchez-Dominguez R.; Garcia-Torralba A.; Alberquilla O.; Lopez-Manzaneda S.; Fananas S.; Nieto V.; Molinos A.; Garcia-Bravo M.; Yanez R.; Bueren J. A.; Navarro S.; Segovia J. C.

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Preclinical biosafety studies of lentiviral vector-mediated gene therapy in erythrocyte pyruvate kinase deficiency

机译：慢病毒载体介导的基因治疗红细胞丙酮酸激酶缺乏的临床前生物安全研究

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《Human gene therapy》 |2018年第12期|共1页
作者
Quintana-Bustamante O.; Sanchez-Dominguez R.; Garcia-Torralba A.; Alberquilla O.; Lopez-Manzaneda S.; Fananas S.; Nieto V.; Molinos A.; Garcia-Bravo M.; Yanez R.; Bueren J. A.; Navarro S.; Segovia J. C.;
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CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

CIEMAT CIBERER IIS FJD Madrid Spain;

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正文语种 eng
中图分类治疗学;
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专利

1. Preclinical biosafety studies of lentiviral vector-mediated gene therapy in erythrocyte pyruvate kinase deficiency [J] . Quintana-Bustamante O., Sanchez-Dominguez R., Garcia-Torralba A., Human gene therapy . 2018,第12期

机译：慢病毒载体介导的基因治疗红细胞丙酮酸激酶缺乏的临床前生物安全研究
2. Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency [J] . Quintana Bustamante Oscar, Fananas-Baquero Sara, Dever Daniel P., Blood: The Journal of the American Society of Hematology . 2018,第NovaelaTreatmentAppr期

机译：高效的CRISPR / CAS9介导的PKLR在人造血祖细胞和干细胞中的基因编辑，用于丙酮酸激酶缺乏的基因治疗
3. Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters. [J] . Hunter MJ, Zhao H, Tuschong LM, Human gene therapy . 2011,第6期

机译：利用鼠干细胞病毒和人磷酸甘油酸激酶启动子的慢病毒载体进行犬白细胞粘附缺乏的基因治疗。
4. The Response of the Metabolic Network of the Red Blood Cell to Pyruvate Kinase Deficiency [C] . Xiaoliang Sun, Zuhong Lu . 2005

机译：红细胞代谢网络对丙酮酸激酶缺乏症的反应
5. Improving lentiviral vector-mediated gene transfer by understanding cellular barriers. [D] . Oakland, Mayumi. 2013

机译：通过了解细胞屏障改善慢病毒载体介导的基因转移。
6. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID [O] . Sabine Charrier, Chantal Lagresle-Peyrou, Valentina Poletti, 2019

机译：临床上适用于Artemis-SCID基因治疗的慢病毒载体的生物安全性研究
7. Estudios preclínicos para el tratamiento mediante terapia génica de la deficiencia de piruvato quinasa. udPreclinical studies for the gene therapy treatment of pyruvate kinase deficiency [O] . García Gómez María 2013

机译：基因治疗丙酮酸激酶缺乏症的临床前研究。您基因治疗丙酮酸激酶缺乏症的临床前研究

Preclinical biosafety studies of lentiviral vector-mediated gene therapy in erythrocyte pyruvate kinase deficiency

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