AAV-mediated gene therapy shows efficacy on the biochemical and neurological phenotype of the nucleoside-enhanced mouse model of MNGIE

被引：0
|
作者：
Vila-Julia, F. [1 ,2 ]
Cabrera-Perez, R. [1 ,2 ]
Lope-Piedrafita, S. [3 ,4 ]
Camara, Y. [1 ,2 ]
Hirano, M. [5 ]
Mingozzi, F. [6 ,7 ,8 ]
Torres-Torronteras, J. [1 ,2 ]
Marti, R. [1 ,2 ]
机构：
[1] Univ Autonoma Barcelona, Vall dHebron Res Inst, Res Grp Neuromuscular & Mitochondrial Dis, Barcelona, Catalonia, Spain
[2] Inst Salud Carlos III, Biomed Network Res Ctr Rare Dis, Madrid, Spain
[3] Univ Autonoma Barcelona, Nucl Magnet Resonance Serv, Cerdanyola Del Valles, Catalonia, Spain
[4] Univ Autonoma Barcelona, Biomed Network Res Ctr Bioengn Biomat & Nanomed, Cerdanyola Del Valles, Catalonia, Spain
[5] Columbia Univ, Dept Neurol, Med Ctr, H Houston Merritt Neuromuscular Res Ctr, New York, NY USA
[6] Genethon, Evry, France
[7] INSERM, U951, Evry, France
[8] Univ Pierre & Marie Curie, Paris, France
来源：
HUMAN GENE THERAPY | 2019年 / 30卷 / 11期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
P442
引用
收藏
页码：A147 / A148
页数：2
相关论文
共 50 条
  • [1] AAV-mediated gene therapy in a mouse model of sialidosis
    van de Vlekkert, Diantha
    Hu, Huimin
    Weesner, Jason A.
    Gomero, Elida M.
    d'Azzo, Alessandra
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 129 - 130
  • [2] Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
    Di Meo, Ivano
    Auricchio, Alberto
    Lamperti, Costanza
    Burlina, Alberto
    Viscomi, Carlo
    Zeviani, Massimo
    EMBO MOLECULAR MEDICINE, 2012, 4 (09) : 1008 - 1014
  • [3] AAV-Mediated Gene Replacement Therapy in Mouse Model of Tuberous Sclerosis
    Prabhakar, Shilpa
    Zhang, Xuan
    Goto, June
    Sena-Esteves, Miguel
    Ramesh, Vijaya
    Bronson, Roderick
    Chen, John W.
    Stemmer-Rachamimov, Anat O.
    Kwiatkowski, David J.
    Breakefield, Xandra O.
    MOLECULAR THERAPY, 2015, 23 : S78 - S78
  • [4] Study on AAV-mediated gene therapy for diabetes in humanized liver mouse to predict efficacy in humans
    Hashimoto, Haruo
    Mizushima, Tomoko
    Ogura, Tomoyuki
    Kagawa, Takahiro
    Tomiyama, Kayo
    Takahashi, Ri-ichi
    Yagoto, Mika
    Kawai, Kenji
    Chijiwa, Tsuyoshi
    Nakamura, Masato
    Suemizu, Hiroshi
    BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2016, 478 (03) : 1254 - 1260
  • [5] AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression
    Gicquel, Evelyne
    Maizonnier, Natacha
    Foltz, Steven J.
    Martin, William J.
    Bourg, Nathalie
    Svinartchouk, Fedor
    Charton, Karine
    Beedle, Aaron M.
    Richard, Isabelle
    HUMAN MOLECULAR GENETICS, 2017, 26 (10) : 1952 - 1965
  • [6] AAV-mediated Stambp gene replacement therapy rescues neurological defects in a mouse model of microcephaly-capillary malformation syndrome
    Hu, Meixin
    Li, Jun
    Deng, Jingxin
    Liu, Chunxue
    Liu, Yingying
    Li, Huiping
    Feng, Weijun
    Xu, Xiu
    MOLECULAR THERAPY, 2024, 32 (11) : 4095 - 4107
  • [7] Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders
    Marrone, Lara
    Marchi, Paolo M.
    Azzouz, Mimoun
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2022, 22 (09) : 1163 - 1176
  • [8] Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
    Zhou, Livia
    Wang, Yafeng
    Xu, Yiran
    Zhang, Yaodong
    Zhu, Changlian
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2024, 32 (04)
  • [9] Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB
    Heldermon, Coy D.
    Ohlemiller, Kevin K.
    Herzog, Erik D.
    Vogler, Carole
    Qin, Elizabeth
    Wozniak, David F.
    Tan, Yun
    Orrock, John L.
    Sands, Mark S.
    MOLECULAR THERAPY, 2010, 18 (05) : 873 - 880
  • [10] AAV-mediated gene therapy for retinal disorders: from mouse to man
    Buch, P. K.
    Bainbridge, J. W.
    Ali, R. R.
    GENE THERAPY, 2008, 15 (11) : 849 - 857
12345