Severe Leukocyte Adhesion Deficiency-I (LAD-I) Lentiviral-Mediated Ex-Vivo Gene Therapy: Ongoing Phase 1/2 Study Results

被引：0
|
作者：
Booth, Claire [1 ]
Sevilla, Julian [2 ,3 ]
Lopez, Maria Chitty [4 ]
Almarza, Elena [5 ]
Zubicaray, Josune [2 ,3 ]
Chetty, Kritika [6 ]
Moore, Theodore [7 ]
Bueren, Juan [3 ,8 ]
Schwartz, Jonathan [4 ]
Kohn, Donald [9 ]
机构：
[1] UCL Great Ormond St Inst Child Hlth, Gene Therapy & Paediat Immunol, London, England
[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] Rocket Pharmaceut Inc, Proc Dev LVV, Cranbury, NJ USA
[6] UCL Great Ormond St Inst Child Hlth, London, England
[7] Univ Calif Los Angeles, Pediat Blood & Marrow Transplant Program, Los Angeles, CA USA
[8] Ctr Invest Energet Medioambient Tecnol CIEMAT, Hematopoiet Innovat Therapies Div, Madrid, Spain
[9] Univ Calif Los Angeles, UCLA Gene & Cell Therapy, Los Angeles, CA USA
来源：
CLINICAL IMMUNOLOGY | 2023年 / 250卷
关键词：
Gene Therapy; Primary Immundodeficiencies; Leukocyte Adhesion Deficiency-I;
D O I：
10.1016/j.clim.2023.109354
中图分类号：
R392 [医学免疫学]; Q939.91 [免疫学];
学科分类号：
100102 ;
摘要：
7
引用
收藏
页数：1
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