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：

引用

页数：1

共 45 条

[1] 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
[2] 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
[3] 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
[4] 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
[5] 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
Booth, C.
Kohn, D. B.
Sevilla, J.
Rao, G. R.
Almarza, E.
Terrazas, D.
Nicoletti, E.
Fernandes, A.
Kuo, C.
De Oliveira, S.
Moore, T. B.
Law, K. M.
Beard, B. C.
Choi, G.
Zeini, M.
Mesa-Nunez, C.
Thrasher, A. J.
Bueren, J.
Schwartz, J.
HUMAN GENE THERAPY, 2021, 32 (19-20) : A5 - A5
[6] 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
[7] 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
[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

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