LENTIVIRAL-MEDIATED GENE THERAPY FOR SEVERE PYRUVATE KINASE DEFICIENCY: RESULTS FROM AN ONGOING GLOBAL PHASE 1 STUDY

被引：0

作者：

Lopez Lorenzo, Jose Luis ^{[1
,2
]}

Shah, Ami ^{[3
]}

Sevilla, Julian ^{[5
,6
]}

Navarro, Susana ^{[2
,6
,7
]}

Llanos, Lucia ^{[1
,2
]}

de Camino Gaisse, Begona Perez ^{[1
,2
]}

Sanchez, Sol ^{[1
,2
]}

Zubicaray, Josune ^{[5
]}

Glader, Bertil ^{[3
,4
]}

Chien, May ^{[3
,4
]}

Quintana Bustamante, Oscar ^{[2
,6
,7
]}

Zeini, Miriam ^{[8
]}

Choi, Grace ^{[8
]}

Nicoletti, Eileen ^{[8
]}

Rao, Gayatri ^{[8
]}

Roncarolo, Maria Grazia ^{[3
,4
]}

Bueren, Juan ^{[2
,6
,7
]}

Schwartz, Jonathan ^{[8
]}

Carlos Segovia, Jose ^{[2
,6
,7
]}

机构：

[1] Hosp Univ Fdn Jimenez Diaz, Madrid, Spain

[2] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Madrid, Spain

[3] Stanford Univ, Sch Med, Stanford, CA USA

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

[5] Hosp Infantil Univ Nino Jesus HIUNJ, Fdn invest Biomed, Madrid, Spain

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

[7] Ctr Invest Energet Medioambientales & Tecnol CIEM, Madrid, Spain

[8] Rocket Pharmaceut Inc, Cranbury, NJ USA

来源：

BONE MARROW TRANSPLANTATION | 2023年 / 58卷 / SUPP1期

关键词：

D O I：

暂无

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

P181

引用

页码：275 / 276

页数：2

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