Interim Results from an Ongoing Global Phase 1 Study of Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency

被引：0
|
作者：
Lopez Lorenzo, J. L. [1 ,2 ]
Shah, A. J. [3 ,4 ,5 ]
Sevilla, J. [6 ,7 ]
Navarro, S. [2 ,7 ,8 ]
Llanos, L. [1 ,2 ]
de Camino Gaisse, B. Perez [1 ,2 ]
Sanchez, S. [1 ]
Zubicaray, J.
Glader, B. [4 ,5 ]
Chien, M. [4 ,5 ]
Bustamante, O. [2 ,7 ,8 ]
Zeini, M.
Choi, G.
Nicoletti, E.
Rao, G. R. [9 ]
Roncarolo, M. G. [4 ]
Bueren, J. A. [2 ,7 ,8 ]
Schwartz, J. D. [9 ]
Segovia, J. C. [2 ]
机构：
[1] Hosp Univ Fdn Jimenez Diaz, Inst Invest Sanit Fdn Jimenez Diaz IIS FJD, Madrid, Spain
[2] Inst Invest Sanit Fdn Jimenez prim, Unidad Mixta Terapias Avanzadas, Madrid, Spain
[3] Stanford Univ, Ctr Definit & Curat Med, Stanford, CA USA
[4] Stanford Univ, Sch Med, Stanford, CA USA
[5] Lucile Packard Childrens Hosp, Palo Alto, CA USA
[6] Hosp Infantil Univ Nino Jesus HIUNJ, Madrid, Spain
[7] Ctr Invest Biomed Red Enfermedades Raras CIBERER, Madrid, Spain
[8] Ctr Invest Energet Medioambient & Tecnol CIEMAT, Madrid, Spain
[9] Rocket Pharmaceut Inc, New York, NY USA
来源：
HUMAN GENE THERAPY | 2022年 / 33卷 / 23-24期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
P128
引用
收藏
页码：A58 / A58
页数：1
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