Development and validation of a novel AAV gene therapy for mucopolysaccharidosis type IIIB in a large animal

被引：0
|
作者：
Banchi, Elena Gaia [1 ]
Alonso, Rafael [1 ]
Jacquot, Sylvie [1 ]
Fransquin, Kevin [1 ]
Courtot, Enora [1 ]
Denis, Timothee De Saint [1 ]
Ausseil, Jerome [2 ]
Piguet, Francoise [1 ]
机构：
[1] Paris Brain Inst, Paris, France
[2] Toulouse Univ Hosp, Toulouse, France
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2023.107768
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
29
引用
收藏
页数：1
相关论文
共 50 条
  • [21] 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
  • [22] 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
  • [23] 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
  • [24] Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
    Roca, Carles
    Motas, Sandra
    Marco, Sara
    Ribera, Albert
    Sanchez, Victor
    Sanchez, Xavier
    Bertolin, Joan
    Leon, Xavier
    Perez, Jennifer
    Garcia, Miguel
    Villacampa, Pilar
    Ruberte, Jesus
    Pujol, Anna
    Haurigot, Virginia
    Bosch, Fatima
    HUMAN MOLECULAR GENETICS, 2017, 26 (08) : 1535 - 1551
  • [25] AAV-mediated gene therapy to treat mucopolysaccharidosis type IVA in a new mouse model of the disease
    Sanchez, V.
    Bertolin, J.
    Ribera, A.
    Sanchez, X.
    Marco, S.
    Motas, S.
    Perez, J.
    Roca, C.
    Elias, G.
    Garcia, M.
    Leon, X.
    Haurigot, V.
    Ruberte, J.
    Bosch, F.
    HUMAN GENE THERAPY, 2019, 30 (11) : A172 - A172
  • [26] Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
    Jung, Sung-Chul
    Park, Eun-Sook
    Choi, Eun Nam
    Kim, Chi Hwa
    Kim, Su Jin
    Jin, Dong-Kyu
    MOLECULES AND CELLS, 2010, 30 (01) : 13 - 18
  • [27] AAV-Mediated Gene Therapy to Treat Mucopolysaccharidosis Type IVA in a New Morquio a Mouse Model
    Sanchez, Victor
    Bertolin, Joan
    Ribera, Albert
    Sanchez, Xavier
    Marco, Sara
    Motas, Sandra
    Perez, Jennifer
    Roca, Carles
    Elias, Gemma
    Garcia, Miquel
    Leon, Xavier
    Haurigot, Virginia
    Ruberte, Jesus
    Bosch, Fatima
    MOLECULAR THERAPY, 2020, 28 (04) : 492 - 492
  • [28] Correction of Neurological Manifestations of Mucopolysaccharidosis IIIC by a Novel Rationally Designed Neurotropic AAV Gene Therapy Vector
    O'Leary, Claire
    Antunes, Andre
    Tordo, Julie
    Liao, Ai Yin
    Gleitz, Helene
    Parker, Helen
    Holley, Rebecca
    Youshani, Amir Saam
    Stacey, Kevin
    Davis, Daniel
    Dridi, Larbi
    Martins, Carla
    Pshezhetsky, Alexey
    Linden, Michael
    Henckaerts, Els
    Bigger, Brian
    MOLECULAR THERAPY, 2017, 25 (05) : 244 - 244
  • [29] Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosis
    McIvor, R. Scott
    Kozarsky, Karen
    Laoharawee, Kanut
    Podetz-Pedersen, Kelly M.
    Kitto, Kelley
    Riedl, Maureen
    Whitley, Chester B.
    Vulchanova, Lucy
    Fairbanks, Carolyn A.
    Frey, William H.
    Low, Walter C.
    Belur, Lalitha R.
    MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S93 - S93
  • [30] Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
    Tardieu, Marc
    Zerah, Michel
    Gougeon, Marie-Lise
    Ausseil, Jerome
    de Bournonville, Stephanie
    Husson, Beatrice
    Zafeiriou, Dimitrios
    Parenti, Giancarlo
    Bourget, Philippe
    Poirier, Beatrice
    Furlan, Valerie
    Artaud, Cecile
    Baugnon, Thomas
    Roujeau, Thomas
    Crystal, Ronald G.
    Meyer, Christian
    Deiva, Kumaran
    Heard, Jean-Michel
    LANCET NEUROLOGY, 2017, 16 (09): : 712 - 720
12345