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

页码：A58 / A58

页数：1

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