Ex-vivo Autologous Haematopoietic Stem Cell Gene Therapy in Mucopolysaccharidosis Type IIIA

被引:0
|
作者
Kinsella, Jane [1 ]
Jones, Simon [2 ]
Church, Heather [3 ]
Jones, Ceri [3 ]
Ellison, Stuart [4 ]
Booth, Claire [5 ]
Buckland, Karen [5 ]
Rico, Diego Leon [5 ]
Bigger, Brian [4 ]
Thrasher, Adrian J. [5 ]
Wynn, Robert [1 ]
机构
[1] MFT, Royal Manchester Childrens Hosp, Manchester, Lancs, England
[2] MFT, Manchester Ctr Genom Med, Manchester, Lancs, England
[3] MFT, Manchester Ctr Genom Med, Manchester, Lancs, England
[4] Univ Manchester, Manchester, Lancs, England
[5] UCL, Great Ormond St Inst Child Hlth, London, England
关键词
D O I
暂无
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
P185
引用
收藏
页码:320 / 320
页数:1
相关论文
共 50 条
  • [31] Skeletal Damage and Cross-correction after ex-vivo Gene Therapy in Mucopolysaccharidosis type I Hurler
    Santi, Ludovica
    Crippa, Stefania
    Capo, Valentina
    Penna, Sara
    Savoia, Evelyn
    De Ponti, Giada
    Alberti, Gaia
    Berti, Margherita
    Ceriotti, Selene
    Consiglieri, Giulia
    Tucci, Francesca
    Riminucci, Mara
    Corsi, Alessandro
    Barbero, Andrea
    Martin, Ivan
    Sanvito, Francesca
    Norata, Rossana
    Rocchi, Martina
    Bolamperti, Simona
    Villa, Isabella
    Aiuti, Alessandro
    Villa, Anna
    Bernardo, Maria Ester
    JOURNAL OF BONE AND MINERAL RESEARCH, 2024, 39 : 30 - 31
  • [32] Ex vivo expansion of haematopoietic stem cells and gene therapy development
    Zubler, Rudolf H.
    SWISS MEDICAL WEEKLY, 2006, 136 (49-50) : 795 - 799
  • [33] Preclinical Development of an Ex Vivo Gene Therapy for Mucopolysaccharidosis Type II
    Das, Sabyasachi
    Rruga, Fatlum
    Rigon, Laura
    Patel, Janki
    Milazzo, Rita
    Poletti, Valentina
    Biffi, Alessandra
    MOLECULAR THERAPY, 2022, 30 (04) : 117 - 117
  • [34] Preclinical Development of an Ex Vivo Gene Therapy for Mucopolysaccharidosis Type II
    Poletti, Valentina
    Das, Sabyasachi
    Rruga, Fatlum
    Dimartino, Agnese
    Montepeloso, Annita
    Patel, Janki
    Ferro, Francesca
    Cavalca, Eleonora
    Politi, Letterio
    Biffi, Alessandra
    MOLECULAR THERAPY, 2023, 31 (04) : 517 - 517
  • [35] Preclinical development of an ex vivo gene therapy for Mucopolysaccharidosis type II
    Poletti, V.
    Rruga, F.
    Das, S.
    Milazzo, R.
    Dimartino, A.
    Spadini, S.
    Politi, L. S.
    Rizzardi, P.
    Vallanti, G.
    Montepeloso, A.
    Biffi, A.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A242 - A243
  • [36] Preclinical development of an ex vivo gene therapy for Mucopolysaccharidosis type II
    Das, S.
    Rruga, F.
    Rigon, L.
    Patel, J.
    Milazzo, R.
    Poletti, V.
    Biffi, A.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A3 - A3
  • [37] 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
  • [38] EX-VIVO PROCEDURES ASSOCIATED TO AUTOLOGOUS HEMATOPOIETIC STEM-CELL TRANSPLANTATION
    FERNANDEZ, M
    MINGUELL, JJ
    REVISTA MEDICA DE CHILE, 1994, 122 (06) : 699 - 704
  • [39] Clinical trial update: ex vivo autologous stem cell gene therapy in MPSIIIA
    Kinsella, J. L.
    Potter, J. E.
    Jones, S. A.
    Thrasher, A. J.
    Booth, C.
    Buckland, K. F.
    Izotova, N.
    Rust, S.
    Weisberg, D.
    Church, H. J.
    Brammeier, K. L.
    Jones, C.
    Lee, H.
    Ford, L.
    Sadegholnejat, A.
    Holley, R.
    Ellison, S.
    Bigger, B.
    Wynn, R. F.
    BONE MARROW TRANSPLANTATION, 2022, 57 (SUPPL 1) : 56 - 57
  • [40] 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