Ex vivo lentiviral gene therapy for mucopolysaccharidosis type IVA

被引：0
|
作者：
Celik, Betul [1 ]
Rintz, Estera [2 ]
Nidhi, Fnu [3 ]
Khan, Shaukat [4 ]
Tomatsu, Shunji [4 ]
机构：
[1] Univ Delaware, Wilmington, DE USA
[2] Univ Gdansk, Gdansk, Poland
[3] Univ Delaware, Newark, DE USA
[4] Neumors Childrens Hosp, Wilmington, DE USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2023.107789
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
50
引用
收藏
页数：1
相关论文
共 50 条
  • [1] Lentiviral gene therapy for mucopolysaccharidosis type IVA
    Celik, Betul
    Rintz, Estera
    Nidhi, Fnu
    Khan, Shaukat
    Tomatsu, Shunji
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 23 - 24
  • [2] 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
  • [3] 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
  • [4] 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
  • [5] 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
  • [6] 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
  • [7] 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
  • [8] Pre-clinical development of an ex-vivo lentiviral-based Hematopoietic Stem/Progenitor Cells (HSPC)-gene therapy (GT) approach for Mucopolysaccharidosis type IVA (MPSIVA)
    Crippa, S.
    Quaranta, P.
    Berti, M.
    Santi, L.
    De Ponti, G.
    Jofra-Hernandez, R.
    Basso-Ricci, L.
    Rilievo, A. A.
    Alberti, G.
    Forni, C.
    Visigalli, I.
    Albertini, P.
    Paoli, A.
    Morrone, A.
    Parini, R.
    Gentner, B.
    Scala, S.
    Aiuti, A.
    Bernardo, M. E.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A170 - A171
  • [9] Ex vivo HSC-based gene therapy for mucopolysaccharidosis type I
    Gentner, B.
    Bernardo, M. E.
    Zonari, E.
    Redaelli, D.
    Meo, O.
    Desantis, G.
    Visigalli, I.
    Cristofori, P.
    la Marca, G.
    Serafini, M.
    Montini, E.
    Aiuti, A.
    HUMAN GENE THERAPY, 2017, 28 (12) : A69 - A69
  • [10] In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors
    Di Natale, P
    Di Domenico, C
    Villani, GRD
    Lombardo, A
    Follenzi, A
    Naldini, L
    EUROPEAN JOURNAL OF BIOCHEMISTRY, 2002, 269 (11): : 2764 - 2771
12345