Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC

被引：0
|
作者：
Badell-Grau, Rafael [1 ]
Pakravesh, Kasra [1 ]
Sivakumar, Anusha [1 ]
Losay, Pauline [1 ]
Duong, Kalvin [1 ]
Tran, Christine [1 ]
Pithia, Rushil [1 ]
Khare, Veenita [1 ]
Thai, Kevin [1 ]
Cherqui, Stephanie [1 ]
机构：
[1] Univ Calif San Diego, San Diego, CA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.107018
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
35
引用
收藏
页码：13 / 13
页数：1
相关论文
共 50 条
  • [41] Neurodevelopmental benefits of early hematopoietic stem cell transplantation in mucopolysaccharidosis type II
    Collyer, John W.
    Rajan, Deepa
    Poe, Michele
    Szabolcs, Paul
    Escolar, Maria L.
    MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02) : 22 - 23
  • [42] Ex vivo lentiviral gene transfer targeting hematopoietic stem cells for therapy of mucopolysaccharidosis type II, Hunter syndrome
    McIvor, R. Scott
    Wolf, Daniel A.
    Podetz-Petersen, Kelly
    Nan, Zhenhong
    Whitley, Chester B.
    Muenzer, Joseph
    Orchard, Paul J.
    Low, Walter C.
    MOLECULAR GENETICS AND METABOLISM, 2009, 96 (02) : S30 - S31
  • [43] Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA
    Ellison, Stuart M.
    Sergijenko, Ana
    Langford-Smith, Alex
    Langford-Smith, Kia
    Wilkinson, Fiona
    Wynn, Rob
    Bigger, Brian
    MOLECULAR GENETICS AND METABOLISM, 2015, 114 (02) : S41 - S41
  • [44] Supraphysiological Enzyme and Early Neurocognitive Outcomes after Autologous Hematopoietic Stem Cell Gene Therapy in Patients with Mucopolysaccharidosis IIIA
    Bigger, Brian W.
    Kinsella, Jane
    Potter, Jane
    Holley, Rebecca
    Searle, Rachel
    Learmonth, Yuko Ishikawa
    Buckland, Karen F.
    Rust, Stewart
    Weisberg, Daniel
    Bromley, Rebecca
    Church, Heather
    Tylee, Karen
    Brammeier, K.
    Lee, H.
    Ford, L.
    Ellison, Stuart M.
    Thrasher, Adrian J.
    Booth, Claire
    Jones, Simon
    Wynn, Robert F.
    MOLECULAR THERAPY, 2023, 31 (04) : 192 - 192
  • [45] Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IH (Hurler Syndrome): An Interim Analysis with a Median Follow Up of 19 Months
    Gentner, Bernhard
    Bernardo, Maria Ester
    Tucci, Francesca
    Fumagalli, Francesca
    Pontesilli, Silvia
    Silvani, Paolo
    Zonari, Erika
    Miglietta, Simona
    Montini, Eugenio
    Calabria, Andrea
    Ciceri, Fabio
    Galimberti, Stefania
    La Marca, Giancarlo
    Parini, Rossella
    Naldini, Luigi
    Aiuti, Alessandro
    MOLECULAR THERAPY, 2021, 29 (04) : 114 - 115
  • [46] Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model
    Celik, Betul
    Rintz, Estera
    Sansanwal, Nidhi
    Khan, Shaukat
    Bigger, Brian
    Tomatsu, Shunji
    HUMAN GENE THERAPY, 2024, 35 (21-22) : 917 - 937
  • [47] Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA
    Ellison, S. M.
    Sergijenko, A.
    Langford-Smith, A.
    Langford-Smith, K.
    Wynn, R.
    Wilkinson, F.
    Bigger, B.
    HUMAN GENE THERAPY, 2015, 26 (10) : A16 - A16
  • [48] Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA
    Ellison, Stuart
    Sergijenko, Ana
    Langford-smith, Alex
    Langford-smith, Kia
    Wynn, Rob
    Wilkinson, Fiona
    Bigger, Brian
    HUMAN GENE THERAPY, 2015, 26 (09) : A27 - A27
  • [49] Brain targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
    Gleitz, H.
    Liao, A.
    Cook, J.
    Rowlston, S.
    Forte, G.
    D'Souza, Z.
    O'Leary, C.
    Holley, R.
    Bigger, B.
    HUMAN GENE THERAPY, 2018, 29 (12) : A28 - A28
  • [50] Hematopoietic Stem Cell Gene Therapy For Mucopolysaccharidosis Type I, Hurler (MPS-IH): Biological Efficacy and Preliminary Evidence of Early Clinical Response
    Bernardo, Maria Ester
    Gentner, Bernhard
    Tucci, Francesca
    Fumagalli, Francesca
    Ciotti, Francesca
    Sarzana, Marina
    Pontesilli, Silvia
    Baldoli, Cristina
    De Pellegrin, Maurizio
    Silvani, Paolo
    Darin, Silvia
    Consiglieri, Giulia
    Filisetti, Chiara
    Miglietta, Simona
    Zonari, Erika
    Parini, Rossella
    Rovelli, Attilio
    La Marca, Giancarlo
    Ciceri, Fabio
    Naldini, Luigi
    Aiuti, Alessandro
    BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 65 - 66
12345