LENTIVIRAL-MEDIATED EX-VIVO GENE THERAPY FOR PEDIATRIC PATIENTS WITH SEVERE LEUKOCYTE ADHESION DEFICIENCY-I (LAD-I): INTERIM RESULTS FROM AN ONGOING PHASE 1/2 STUDY

被引：0

作者：

Kohn, Donald ^{[1
]}

Sevilla, Julian ^{[2
,3
]}

Rao, Gayatri ^{[4
]}

Lopez, Maria Chitty ^{[4
]}

Almarza, Elena ^{[4
]}

Terrazas, Dayna ^{[1
]}

Zubicaray, Josune ^{[2
,3
]}

Gonzalez-Vicent, Marta ^{[2
,3
]}

Chetty, Kritika ^{[5
]}

O'Toole, Grainne ^{[5
]}

Xu-Bayford, Jinhua ^{[5
]}

Nicoletti, Eileen ^{[4
]}

Fernandes, Augustine ^{[1
]}

Kuo, Caroline ^{[1
]}

De Oliveira, Satiro ^{[1
]}

Moore, Theodore B. ^{[1
]}

Choi, Grace ^{[4
]}

Zeini, Miriam ^{[4
]}

Mesa-Nunez, Cristina ^{[4
]}

Thrasher, Adrian ^{[5
]}

Bueren, Juan ^{[3
,6
]}

Schwartz, Jonathan ^{[4
,7
]}

Booth, Claire ^{[5
]}

机构：

[1] Univ Calif Los Angeles, Los Angeles, CA USA

[2] Hosp Infantil Univ Nino Jesus HIUNJ, Fdn Invest Biomed, Madrid, Spain

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

[4] Rocket Pharmaceut Inc, Cranbury, NJ USA

[5] UCL Great Ormond St Inst Child Hlth, London, England

[6] Ctr Invest Energet Medioambient & Tecnol CIEMAT, Madrid, Spain

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

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：

O061

引用

页码：61 / 62

页数：2

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