A phase 1/2 study of lentiviral-mediated ex-vivo gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I): Interim results

被引：0
|
作者：
Booth, C. [1 ]
Kohn, D. B. [2 ]
Sevilla, J. [3 ,4 ]
Rao, G. R. [5 ]
Almarza, E. [4 ,5 ,6 ]
Terrazas, D. [2 ]
Nicoletti, E. [5 ]
Fernandes, A. [2 ]
Kuo, C. [2 ]
De Oliveira, S. [2 ]
Moore, T. B. [2 ]
Law, K. M. [5 ]
Beard, B. C. [5 ]
Choi, G. [5 ]
Zeini, M. [5 ]
Mesa-Nunez, C. [4 ,6 ,7 ]
Thrasher, A. J. [1 ]
Bueren, J. [4 ,6 ,7 ]
Schwartz, J. [5 ]
机构：
[1] UCL Great Ormond St Hosp GOSH, Inst Child Hlth, Infect Immun & Inflammat Dept, London, England
[2] Univ Calif Los Angeles, Los Angeles, CA USA
[3] Hosp Infantil Univ Nino Jesus, Hematol & Hemoterapia, Fdn Invest Biomed HIUNJ, Madrid, Spain
[4] Ctr Invest Biomed Red Enfermedades Raras CIBERER, Madrid, Spain
[5] Rocket Pharmaceut Inc, Cranbury, NJ USA
[6] Ctr Invest Energet Medioambientales & Tecnol CIEM, Div Hematopoiet Innovat Therapies, Madrid, Spain
[7] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD UA, Adv Therapies Unit, Madrid, Spain
来源：
HUMAN GENE THERAPY | 2021年 / 32卷 / 19-20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：
OR09
引用
收藏
页码：A5 / A5
页数：1
相关论文
共 40 条
  • [1] A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
    Kohn, Donald
    Rao, Gayatri
    Almarza, Elena
    Terrazas, Dayna
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    De Oliveira, Satiro
    Moore, Theodore
    Law, Ken
    Beard, Brian
    Sevilla, Julian
    Mesa-Nunez, Cristina
    Booth, Claire
    Thrasher, Adrian
    Bueren, Juan
    Schwartz, Jonathan
    JOURNAL OF CLINICAL IMMUNOLOGY, 2021, 41 (SUPPL 1) : S126 - S126
  • [2] A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
    Kohn, Donald B.
    Booth, Claire
    Sevilla, Julian
    Rao, Gayathri R.
    Almarza, Elena
    Terrazas, Dayna
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    Oliveira, Satirode
    Moore, Theodore B.
    Law, Kenneth M.
    Beard, Brian C.
    Choi, Grace
    Zeini, Miriam
    Mesa-Nunez, Cristina
    Thrasher, Adrian J.
    Bueren, Juan A.
    Schwartz, Jonathan D.
    BLOOD, 2021, 138
  • [3] A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
    Kohn, Donald B.
    Rao, Gayatri
    Almarza, Elena
    Terrazas, Dayna
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    De Oliviera, Satiro
    Moore, Theodore
    Law, Ken
    Beard, Brian
    Sevilla, Julian
    Mesa-Nunez, Cristina
    Booth, Claire
    Thrasher, Adrian
    Bueren, Juan
    Schwartz, Jonathan
    MOLECULAR THERAPY, 2021, 29 (04) : 19 - 20
  • [4] Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
    Kohn, D. B.
    Sevilla, J.
    Rao, G.
    Chitty Lopez, M.
    Almarza, E.
    Terrazas, D.
    Zubicaray, J.
    Gonzalez-Vicent, M.
    Chetty, K.
    O'Toole, G.
    Xu-Bayford, J.
    Nicoletti, E.
    Fernandes, A.
    Kuo, C.
    de Oliveira, S.
    Moore, T. B.
    Choi, G.
    Zeini, M.
    Mesa-Nunez, C.
    Thrasher, A. J.
    Bueren, J.
    Schwartz, J.
    Booth, C.
    MOLECULAR THERAPY, 2022, 30 (04) : 550 - 550
  • [5] Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
    Booth, Claire
    Sevilla, Julian
    Rao, Gayatri R.
    Lopez, Maria Chitty
    Almarza, Elena
    Terrazas, Dayna
    Zubicaray, Josune
    Gonzalez Vicent, Marta
    Chetty, Kritika
    O'Toole, Grainne
    Xu-Bayford, Jinhua
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    de Oliveira, Satiro
    Moore, Theodore B.
    Choi, Grace
    Zeini, Miriam
    Mesa-Nunez, Cristina
    Thrasher, Adrian J.
    Bueren, Juan A.
    Schwartz, Jonathan D.
    Kohn, Donald B.
    BLOOD, 2022, 140 : 7774 - 7775
  • [6] 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
    Kohn, Donald
    Sevilla, Julian
    Rao, Gayatri
    Lopez, Maria Chitty
    Almarza, Elena
    Terrazas, Dayna
    Zubicaray, Josune
    Gonzalez-Vicent, Marta
    Chetty, Kritika
    O'Toole, Grainne
    Xu-Bayford, Jinhua
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    De Oliveira, Satiro
    Moore, Theodore B.
    Choi, Grace
    Zeini, Miriam
    Mesa-Nunez, Cristina
    Thrasher, Adrian
    Bueren, Juan
    Schwartz, Jonathan
    Booth, Claire
    BONE MARROW TRANSPLANTATION, 2023, 58 (SUPP1) : 61 - 62
  • [7] Severe Leukocyte Adhesion Deficiency-I (LAD-I) Lentiviral-Mediated Ex-Vivo Gene Therapy: Ongoing Phase 1/2 Study Results
    Booth, Claire
    Sevilla, Julian
    Lopez, Maria Chitty
    Almarza, Elena
    Zubicaray, Josune
    Chetty, Kritika
    Moore, Theodore
    Bueren, Juan
    Schwartz, Jonathan
    Kohn, Donald
    CLINICAL IMMUNOLOGY, 2023, 250
  • [8] A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1
    Kohn, Donald B.
    Rao, Gayatri R.
    Almarza, Elena
    Terrazas, Dayna
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    De Oliveira, Satiro N.
    Moore, Theodore B.
    Law, Kenneth M.
    Beard, Brian C.
    Sevilla, Julian
    Mesa-Nunez, Cristina
    Bueren, Juan A.
    Schwartz, Jonathan D.
    BLOOD, 2020, 136
  • [9] Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study
    Kohn, Donald B.
    Sevilla, Julian
    Rao, Gayatri
    Lopez, Maria Chitty
    Almarza, Elena
    Terrazas, Dayna
    Zubicaray, Josune
    Gonzalez-Vicent, Marta
    Chetty, Kritika
    O'Toole, Grainne
    Xu-Bayford, Jinhua
    Nicoletti, Eileen
    Fernandez, Augustine
    Kuo, Caroline Y.
    De Oliveira, Satiro N.
    Moore, Theodore B.
    Choi, Grace
    Zeini, Miriam
    Mesa-Nunez, Cristina
    Thrasher, Adrian J.
    Bueren, Juan A.
    Schwartz, Jonathan
    Booth, Claire
    MOLECULAR THERAPY, 2023, 31 (04) : 727 - 728
  • [10] A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient
    Kohn, Donald
    Rao, Gayatri
    Almarza, Elena
    Terrazas, Dayna
    Nicoletti, Eileen
    Fernandes, Augustine
    Kuo, Caroline
    De Oliveira, Satiro
    Moore, Theodore
    Law, Ken
    Beard, Brian
    Sevilla, Julian
    Mesa, Christina
    Bueren, Juan
    Schwartz, Jonathan
    JOURNAL OF CLINICAL IMMUNOLOGY, 2020, 40 (SUPPL 1) : S103 - S103
1234