Phase 1/2 clinical trial of autologous hematopoietic stem and progenitor cell (HSPC) gene therapy for cystinosis

被引：0
|
作者：
Cherqui, Stephanie [1 ]
Ball, Edward [2 ]
Del Parigi, Angelo [3 ]
Kohn, Donald [4 ]
Barshop, Bruce [2 ]
机构：
[1] Univ Calif San Diego, Pediat, La Jolla, CA USA
[2] Univ Calif San Diego, La Jolla, CA USA
[3] AVROBIO, Cambridge, MA USA
[4] Univ Calif Los Angeles, Los Angeles, CA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.107055
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
72
引用
收藏
页码：26 / 26
页数：1
相关论文
共 50 条
  • [31] Autologous hematopoietic stem cell transplantation in multiple sclerosis A phase II trial
    Mancardi, Giovanni L.
    Sormani, Maria P.
    Gualandi, Francesca
    Saiz, Albert
    Carreras, Eric
    Merelli, Elisa
    Donelli, Amedea
    Lugaresi, Alessandra
    Di Bartolomeo, Paolo
    Rottoli, Maria R.
    Rambaldi, Alessandro
    Amato, Maria P.
    Massacesi, Luca
    Di Gioia, Massimo
    Vuolo, Luisa
    Curro, Daniela
    Roccatagliata, Luca
    Filippi, Massimo
    Aguglia, Umberto
    Iacopino, Pasquale
    Farge, Dominique
    Saccardi, Riccardo
    NEUROLOGY, 2015, 84 (10) : 981 - 988
  • [32] Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial
    Kinsella, Jane Louise
    Jones, Simon
    Thrasher, Adrian J.
    Booth, Claire
    Buckland, Karen F.
    Izotova, Natalia
    Rust, Stewart
    Weisberg, Daniel
    Church, Heather J.
    Tylee, Karen L.
    Brammeier, Kathryn L.
    Jones, Ceri
    Lee, Helena
    Ford, Laura
    Sadegholnejat, Atusa
    Ellison, Stuart
    Bigger, Brian W.
    Wynn, Robert F.
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S56 - S57
  • [33] Clinical benefit of PD-L1 hematopoietic stem and progenitor cell gene therapy in an animal model of multiple sclerosis
    Milazzo, R.
    Spadini, S.
    Santinon, G.
    Ciervo, Y.
    Kiyemba, M.
    Rigon, L.
    Accardo, M.
    Poletti, V.
    Peviani, M.
    Ben Nasr, M.
    Rizzardi, G. P.
    Fiorina, P.
    Biffi, A.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A15 - A15
  • [34] Clinical Trial Update: EX-VIVO Autologous Stem Cell Gene Therapy in MPSIIIA
    Kinsella, Jane Louise
    Jones, Simon A.
    Thrasher, Adrian J.
    Booth, Claire
    Buckland, Karen F.
    Izotova, Natalia
    Rust, Stewart
    Weisberg, Daniel
    Church, Heather J.
    Tylee, Karen
    Brammeier, Kathryn L.
    Jones, Ceri
    Lee, Helena
    Ford, Laura
    Sadegholnejat, Atusa
    Holley, Rebecca J.
    Ellison, Stuart
    Bigger, Brian
    Wynn, Robert F.
    BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 68 - 69
  • [35] Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
    Gentner, B.
    Tucci, F.
    Galimberti, S.
    Fumagalli, F.
    De Pellegrin, M.
    Silvani, P.
    Camesasca, C.
    Pontesilli, S.
    Darin, S.
    Ciotti, F.
    Sarzana, M.
    Consiglieri, G.
    Filisetti, C.
    Forni, G.
    Passerini, L.
    Tomasoni, D.
    Cesana, D.
    Calabria, A.
    Spinozzi, G.
    Cicalese, M-P
    Calbi, V
    Migliavacca, M.
    Barzaghi, F.
    Ferrua, F.
    Gallo, V
    Miglietta, S.
    Zonari, E.
    Cheruku, P. S.
    Forni, C.
    Facchini, M.
    Corti, A.
    Gabaldo, M.
    Zancan, S.
    Gasperini, S.
    Rovelli, A.
    Boelens, J. J.
    Jones, S. A.
    Wynn, R.
    Baldoli, C.
    Montini, E.
    Gregori, S.
    Ciceri, F.
    Valsecchi, M. G.
    la Marca, G.
    Parini, R.
    Naldini, L.
    Aiuti, A.
    Bernardo, M-E
    NEW ENGLAND JOURNAL OF MEDICINE, 2021, 385 (21): : 1929 - 1940
  • [36] A Novel Highly Conserved Hematopoietic Stem/Progenitor Cell Population for Stem Cell Transplantation and Gene Therapy
    Radtke, Stefan
    Chan, Yan-Yi
    Giese, Morgan A.
    Rongvaux, Anthony
    Adair, Jennifer
    Kiem, Hans-Peter
    BLOOD, 2017, 130
  • [37] Phase 1 Clinical Trial of Autologous Mesenchymal Stem Cell Transplantation in Multiple Sclerosis
    Cohen, Jeffrey
    Bar-Or, Amit
    Bermel, Robert
    Fisher, Elizabeth
    Fox, Robert
    Gerson, Stanton
    Imrey, Peter
    Lazarus, Hilliard
    Planchon, Sarah
    Reese, Jane
    Schwanger, Cynthia
    Skaramagas, Thomai
    NEUROLOGY, 2012, 78
  • [38] Phase 1 Clinical Trial of Autologous Mesenchymal Stem Cell Transplantation in Multiple Sclerosis
    Cohen, Jeffrey
    Bar-Or, Amit
    Bermel, Robert
    Fisher, Elizabeth
    Fox, Robert
    Gerson, Stanton
    Imrey, Peter
    Lazarus, Hilliard
    Planchon, Sarah
    Reese, Jane
    Schwanger, Cynthia
    Skaramagas, Thomai
    NEUROLOGY, 2012, 78
  • [39] Hematopoietic Stem Cell Gene Therapy: Progress on the Clinical Front
    Cavazzana, Marina
    HUMAN GENE THERAPY, 2014, 25 (03) : 165 - 170
  • [40] Enrichment of Human Hematopoietic Stem/Progenitor Cells Facilitates Transduction for Stem Cell Gene Therapy
    Baldwin, Kismet
    Urbinati, Fabrizia
    Romero, Zulema
    Campo-Fernandez, Beatriz
    Kaufman, Michael L.
    Cooper, Aaron R.
    Masiuk, Katelyn
    Hollis, Roger P.
    Kohn, Donald B.
    STEM CELLS, 2015, 33 (05) : 1532 - 1542
12345