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

[41] Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)
Walters, Mark C.
Rasko, John
Hongeng, Suradej
Kwiatkowski, Janet
Schiller, Gary J.
Kletzel, Morris
Ho, P. Joy
Vichinsky, Elliott
von Kalle, Christof
Cavazzana, Marina
Leboulch, Philippe
Petrusich, Alexandria
Soni, Sandeep
Thompson, Alexis A.
BLOOD, 2015, 126 (23)
[42] The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease
Jacobsen, Leslie
Kerner, Julie A.
Ciotti, Rachel
Golipour, Azadeh
Yang, Josie
Pfeifer, Richard
Khan, Aneal
Mason, Chris
MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S51 - S52
[43] The GuardOne clinical trial: a first in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease
Ridha, E.
Jones, J. J.
Abrahamsen, I.
Golipour, A.
Pfeifer, R.
Khan, A.
Mason, C.
Escolar, D.
HUMAN GENE THERAPY, 2021, 32 (19-20) : A80 - A80
[44] Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan
Mamcarz, Ewelina
Zhou, Sheng
Lockey, Timothy
Abdelsamed, Hossam
Cross, Shane
Ma, Zhijun
Dowdy, Jola
Triplett, Brandon
Weiss, Mitchell J.
Ryu, Byoung Y.
Dokmeci, Elif
Aldave, Juan Carlos
De Ravin, Suk See
Youngblood, Benjamin
Long-Boyle, Janel R.
Meagher, Michael M.
Malech, Harry L.
Puck, Jennifer
Cowan, Morton Jerome
Sorrentino, Brian
BLOOD, 2017, 130
[45] Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q Globin Vector (LentiGlobin BB305 Drug Product)
Thompson, Alexis A.
Rasko, John E.
Hongeng, Suradej
Kwiatkowski, Janet L.
Schiller, Gary
von Kalle, Christof
Cavazzana, Marina
Leboulch, Philippe
Petrusich, Alexandria
Soni, Sandeep
Walters, Mark C.
BLOOD, 2014, 124 (21)

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