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
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
  • [1] Phase 1/2 Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2023, 31 (04) : 100 - 100
  • [2] Hematopoietic Stem Cell Gene Therapy for Cystinosis: Updated Results from a Phase 1/2 Clinical Trial
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2022, 30 (04) : 29 - 29
  • [3] HEMATOPOIETIC STEM CELL GENE THERAPY FOR CYSTINOSIS (PHASE I/II CLINICAL TRIAL): UPDATED RESULTS
    Barshop, Bruce A.
    Ball, Edward
    Dohil, Magdalene
    Dohil, Ranjan
    Fiorini, Claudia
    Biasco, Luca
    Kohn, Donald
    Afshari, Natalie
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (04) : 249 - 249
  • [4] Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease
    Urbinati, Fabrizia
    Wherley, Jennifer
    Geiger, Sabine
    Fernandez, Beatriz Campo
    Kaufman, Michael L.
    Cooper, Aaron
    Romero, Zulema
    Marchioni, Filippo
    Reeves, Lilith
    Read, Elizabeth
    Nowicki, Barbara
    Grassman, Elke
    Viswanathan, Shivkumar
    Wang, Xiaoyan
    Hollis, Roger P.
    Kohn, Donald B.
    CYTOTHERAPY, 2017, 19 (09) : 1096 - 1112
  • [5] Hematopoietic Stem Cell Gene Therapy for Cystinosis: Updated Results from a Phase I/II Clinical Trial
    Cherqui, Stephanie
    Barshop, Bruce
    Ball, Edward
    Dohil, Magdalene
    Dohil, Rajan
    Kohn, Donald
    Afshari, Natalie
    MOLECULAR THERAPY, 2021, 29 (04) : 243 - 243
  • [6] Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S25 - S25
  • [7] Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S30 - S30
  • [8] Hematopoietic Stem Cell Gene Therapy for Cystinosis: Initial Results from a Phase I/II Clinical Trial
    Barshop, Bruce
    Ball, Edward
    Dohil, Magdalene
    Kohn, Donald
    Dohil, Rajan
    Benador, Nadine
    Mak, Robert
    Afshari, Natalie
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2020, 28 (04) : 233 - 233
  • [9] Hematopoietic Stem Cell Gene Therapy for Cystinosis
    Ur, Sarah
    Rocca, Celine J.
    Sharma, Jay
    Kohn, Donald B.
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2013, 21 : S190 - S191
  • [10] Towards a phase I clinical trial for autologous hematopoietic stem cells transplantation in cystinosis
    Lobry, Tatiana
    Sharma, Jay
    Ur, Sarah
    Lau, Athena
    Rocca, Celine
    Kohn, Donald B.
    Carbonaro, Denise
    Cabrera, Betty
    Hernandez, Laura
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S86 - S86