Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301

被引：0

作者：

Shah, Ami ^{[1
,2
,3
]}

Sevilla, Julian ^{[4
,5
]}

Lopez Lorenzo, Jose Luis ^{[6
,7
]}

Navarro, Susana ^{[5
,7
,8
]}

Llanos, Lucia ^{[6
,7
]}

de Camino Gaisse, Belgona Perez ^{[6
,7
]}

Sanchez, Sol ^{[6
,7
]}

Zubicaray, Josune ^{[4
]}

Glader, Bert ^{[2
,3
]}

Chien, May ^{[2
,3
]}

Quintana Bustamante, Oscar ^{[5
,7
,8
]}

Gonzalez Vicent, Marta ^{[4
,5
]}

Iriondo, June ^{[4
,5
]}

Rothe, Michael ^{[9
]}

John-Neek, Philipp ^{[9
]}

Bastone, Antonella ^{[9
]}

Schambach, Axel ^{[9
]}

Zeini, Miriam ^{[10
]}

Choi, Grace ^{[10
]}

Nicoletti, Eileen ^{[10
]}

Rao, Gayatri ^{[10
]}

Roncarolo, Maria-Grazia ^{[1
,2
,3
]}

Bueren, Juan A. ^{[5
,7
,8
]}

Schwartz, Jonathan ^{[10
]}

Segovia, Jose-Carlos ^{[5
,7
,8
]}

机构：

[1] Stanford Univ, Ctr Definit & Curat Med, Stanford, CA USA

[2] Stanford Univ, Sch Med, Div Hematol Oncol Stem Cell Transplant Regenerat, Dept Pediat, Stanford, CA USA

[3] Lucile Packard Childrens Hosp, Palo Alto, CA USA

[4] HIUNJ, Fdn Invest Biomed, Hematol & Hemoterapia, Madrid, Spain

[5] Ctr Invest Biomed Red Enfermedades Raras CIBERER, Madrid, Spain

[6] Hosp Univ Fdn Jimenez Diaz, Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Madrid, Spain

[7] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Unidad Mixta Terapias Avanzadas, Madrid, Spain

[8] Ctr Invest Energet Medioambientales & Tecnol CIEM, Unidad Innovac Biomed, Madrid, Spain

[9] Hannover Med Sch, Hannover, Germany

[10] Rocket Pharmaceut, Cranbury, NJ USA

来源：

MOLECULAR THERAPY | 2024年 / 32卷 / 04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：

引用

页码：2 / 3

页数：2

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