Lentiviral Vector Mediated Haematopoietic Stem Cell Gene Therapy for the Treatment of Lysosomal Disease

被引:0
作者
McDermott, J. H. [1 ]
Langford-Smith, A. [1 ]
Sergijenko, A. [1 ]
Langford-Smith, K. J. [1 ]
Liao, A. [1 ]
Jones, S. A. [2 ]
Wraith, J. E. [2 ]
Wynn, R. F. [3 ]
Wilkinson, F. W. [1 ]
Bigger, B. W. [1 ]
机构
[1] Univ Manchester, Manchester, Lancs, England
[2] St Marys Hosp, Manchester M13 0JH, Lancs, England
[3] RMCH, Blood & Marrow Transplant Unit, Manchester, Lancs, England
关键词
D O I
暂无
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
引用
收藏
页码:20 / 20
页数:1
相关论文
共 50 条
[31]   LENTIVIRAL HAEMATOPOIETIC STEM CELL GENE THERAPY (ATIDARSAGENE AUTOTEMCEL) FOR LATE JUVENILE METACHROMATIC LEUKODYSTROPHY (MLD) [J].
Gallo, Vera ;
Calbi, Valeria ;
De Mattia, Fabiola ;
Zambon, Alberto ;
Recupero, Salvatore ;
Fratini, Elena ;
Ciotti, Francesca ;
Fraschini, Maddalena ;
Sangalli, Mara ;
Sarzana, Marina ;
Clerici, Alessandra ;
Morena, Francesco ;
Martino, Sabata ;
Brooks, Jean ;
Richardson, Alan ;
Campbell, Laura ;
Bernardo, Maria Ester ;
Aiuti, Alessandro ;
Fumagalli, Francesca .
BONE MARROW TRANSPLANTATION, 2024, 59 :95-96
[32]   Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in X-linked Adrenoleukodystrophy [J].
Cartier, N. ;
Hacein-Bey-Abina, S. ;
Bartholomae, C. ;
Veres, G. ;
Schmidt, M. ;
Kutschera, I ;
Vidaud, M. ;
Dal-Cortivo, L. ;
Caccavelli, L. ;
Malhaoui, N. ;
Kiermer, V ;
Mittelstaedt, D. ;
Bellesme, C. ;
Audat, F. ;
Blanche, S. ;
Audit, M. ;
l'Homme, B. ;
Bougneres, P. ;
Fischer, A. ;
Von Kalle, C. ;
Cavazzana-Calvo, M. ;
Aubourg, P. .
HUMAN GENE THERAPY, 2010, 21 (06) :765-765
[33]   Hematopoietic stem cell gene therapy with lentiviral vector in X-linked adrenoleukodystrophy [J].
Cartier, N. ;
Hacein-Bey-Abina, S. ;
Bartholomae, C. ;
Schmidt, M. ;
Kutschera, I. ;
Vidaud, M. ;
Dal-Cortivo, L. ;
Malhaoui, N. ;
Bellesme, C. ;
Lefrere, F. ;
l'Homme, B. ;
Bougneres, P. ;
Fischer, A. ;
Von Kalle, C. ;
Cavazzana-Calvo, M. ;
Aubourg, P. .
HUMAN GENE THERAPY, 2010, 21 (10) :1367-1368
[34]   Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].
Cartier, Nathalie ;
Hacein-Bey-Abina, Salima ;
Bartholomae, Cynthia C. ;
Veres, Gabor ;
Schmidt, Manfred ;
Kutschera, Ina ;
Vidaud, Michel ;
Abel, Ulrich ;
Dal-Cortivo, Liliane ;
Caccavelli, Laure ;
Mahlaoui, Nizar ;
Kiermer, Veronique ;
Mittelstaedt, Denice ;
Bellesme, Celine ;
Lahlou, Najiba ;
Lefrere, Francois ;
Blanche, Stephane ;
Audit, Muriel ;
Payen, Emmanuel ;
Leboulch, Philippe ;
l'Homme, Bruno ;
Bougneres, Pierre ;
Von Kalle, Christof ;
Fischer, Alain ;
Cavazzana-Calvo, Marina ;
Aubourg, Patrick .
SCIENCE, 2009, 326 (5954) :818-823
[35]   Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].
Aubourg, Patrick ;
Hacein-Bey-Abina, Salima ;
Bartholomae, Cynthia ;
Schmidt, Manfred ;
Kutschera, Ina ;
Vidaud, Michel ;
Dal-Cortivo, Liliane ;
Mahlaoui, Nizar ;
Frange, Pierre ;
Bellesme, Celine ;
Lefrere, Francois ;
Dufayet, Gaelle ;
Fischer, Alain ;
von Kalle, Christof ;
Cavazzana-Calvo, Marina ;
Cartier, Nathalie .
BLOOD, 2011, 118 (21) :80-80
[36]   A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease [J].
Hart, Kevyn L. ;
Liu, Boya ;
Brown, Devin ;
Campo-Fernandez, Beatriz ;
Tam, Kevin ;
Orr, Katherine ;
Hollis, Roger P. ;
Brendel, Christian ;
Williams, David A. ;
Kohn, Donald B. .
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2024, 32 (02)
[37]   Targeting the blood brain barrier for haematopoietic stem cell gene therapy in neurological lysosomal storage disorders [J].
Critchley, B. J. ;
Naseem, A. ;
Yeung, J. ;
Panchal, N. ;
Seijas, A. Barroeta ;
Santilli, G. ;
Gaspar, H. B. ;
Benedetti, S. .
HUMAN GENE THERAPY, 2022, 33 (23-24) :A167-A168
[38]   Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease [J].
Stok, Merel ;
de Boer, Helen ;
Huston, Marshall W. ;
Jacobs, Edwin H. ;
Roovers, Onno ;
Visser, Trudi P. ;
Jahr, Holger ;
Duncker, Dirk J. ;
van Deel, Elza D. ;
Reuser, Arnold J. J. ;
van Til, Niek P. ;
Wagemaker, Gerard .
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 :1014-1025
[39]   Gene therapy for lysosomal storage diseases using an enhanced lentiviral vector delivery system [J].
Alonso-Ferrero, M. E. ;
Burke, D. ;
Heales, S. ;
Thrasher, A. ;
Gaspar, H. B. .
HUMAN GENE THERAPY, 2011, 22 (10) :A44-A45
[40]   Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].
Cavazzana-Calvo, Marina ;
Cartier, Nathalie ;
Abina, Salima Hacein-Bey ;
Veres, Gabor ;
Schmidt, Manfred ;
Bartholomae, Cynthia ;
Vidaud, Michel ;
Dal-Cortivo, Liliane ;
Caccavelli, Laure ;
Malhaoui, Nizar ;
Frange, Pierre ;
Kiermer, Veronique ;
Mittelstaedt, Denice ;
Simmons, Andrew ;
Bellesme, Celine ;
Lefrere, Francois ;
Lagresle, Chantale ;
Blanche, Stephane ;
Charneau, Pierre ;
Audit, Muriel ;
L'Homme, Bruno ;
Zhao-Emonet, Jing Chao ;
Fichelson, Serge ;
Pflumino, Francoise ;
Dubart-Kupperschmitt, Anne ;
Salzman, Rachel ;
Salzman, Amber ;
Bougneres, Pierre ;
Von Kalle, Christof ;
Fischer, Alain ;
Aubourg, Patrick .
BLOOD, 2008, 112 (11) :304-305