Demonstration of the Feasibility of AAV Vector-Mediated Gene Therapy for Alport Syndrome Using a Newly Established Floxed Mouse Model

被引：0
|
作者：
Das, Ranjan [1 ]
Furusho, Taisuke [1 ]
Sairavi, Anusha [1 ]
Hakui, Hideyuki [1 ]
Luo, Shuhua [1 ]
Li, Lena [1 ]
Miner, Jeffrey H. [2 ]
Nakai, Hiroyuki [1 ,3 ]
机构：
[1] Oregon Hlth & Sci Univ, Mol & Med Genet, Portland, OR USA
[2] Washington Univ, Div Nephrol, Sch Med, St Louis, MO USA
[3] Oregon Hlth & Sci Univ, Mol Microbiol & Immunol, Portland, OR USA
来源：
MOLECULAR THERAPY | 2024年 / 32卷 / 04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：
1584
引用
收藏
页码：739 / 740
页数：2
相关论文
共 50 条
  • [21] Assessment of Toxicity and Biodistribution of an AAV2/8 Vector-Mediated Immunomodulatory Gene Therapy in Mice With Pompe Disease
    Han, Sang-oh
    Wang, Gensheng
    Young, Sarah
    Bali, Deeksha
    Benson, Janet
    Koeberl, Dwight
    MOLECULAR THERAPY, 2014, 22 : S19 - S19
  • [22] Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B
    Lau, Samantha C.
    Grati, Mhamed
    Isgrig, Kevin
    Sinan, Moaz
    Calabro, Kaitlyn R.
    Zhu, Jianliang
    Ishibashi, Yasuko
    Ozgur, Zeynep
    Wafa, Talah
    Belyantseva, Inna A.
    Fitzgerald, Tracy
    Friedman, Thomas B.
    Boye, Sanford L.
    Boye, Shannon E.
    Chien, Wade W.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2023, 30 : 534 - 545
  • [23] Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy
    Hernandez, Raisa Jofra
    Calabria, Andrea
    Sanvito, Francesca
    De Mattia, Fabiola
    Farinelli, Giada
    Scala, Serena
    Visigalli, Ilaria
    Carriglio, Nicola
    De Simone, Maura
    Vezzoli, Michela
    Cecere, Francesca
    Migliavacca, Maddalena
    Basso-Ricci, Luca
    Omrani, Maryam
    Benedicenti, Fabrizio
    Norata, Rossana
    Rancoita, Paola Maria Vittoria
    Di Serio, Clelia
    Albertini, Paola
    Cristofori, Patrizia
    Naldini, Luigi
    Gentner, Bernhard
    Montini, Eugenio
    Aiuti, Alessandro
    Mortellaro, Alessandra
    MOLECULAR THERAPY, 2021, 29 (01) : 86 - 102
  • [24] AAV vector-mediated muscle directed gene therapy results in long-term enzymatic and functional correction in cardio-vascular system of Fabry mouse
    Takahashi, H
    Seino, Y
    Fukumoto, H
    Hirai, Y
    Shimada, T
    Takano, T
    CIRCULATION, 2002, 106 (19) : 28 - 28
  • [25] Retroviral replicating vector-mediated prodrug activator gene therapy in an experimental model of human osteosarcoma
    Kubo, S.
    Takagi-Kimura, M.
    Kasahara, N.
    HUMAN GENE THERAPY, 2017, 28 (12) : A28 - A29
  • [26] Multi-Parametric MRI at 14T for Muscular Dystrophy Mice Treated with AAV Vector-Mediated Gene Therapy
    Park, Joshua
    Wicki, Jacqueline
    Knoblaugh, Sue E.
    Chamberlain, Jeffrey S.
    Lee, Donghoon
    PLOS ONE, 2015, 10 (04):
  • [27] ADENO-ASSOCIATED VIRUS VECTOR-MEDIATED LIVER GENE THERAPY FOR CRIGLER-NAJJAR SYNDROME
    Bosma, P.
    Labrune, P.
    Brunetti-Pierri, N.
    D'Antiga, L.
    Ott, M.
    Beinat, M.
    Ronchi, F.
    Mavilio, F.
    Beuers, U.
    Muro, A.
    Baumann, U.
    Mingozzi, F.
    JOURNAL OF HEPATOLOGY, 2015, 62 : S812 - S812
  • [28] Endostatin gene therapy on murine lung metastases model utilizing cationic vector-mediated intravenous gene delivery
    Y Nakashima
    M Yano
    Y Kobayashi
    S Moriyama
    H Sasaki
    T Toyama
    H Yamashita
    I Fukai
    H Iwase
    Y Yamakawa
    Y Fujii
    Gene Therapy, 2003, 10 : 123 - 130
  • [29] AAV vector-mediated enzyme replacement therapy is efficacious in reversing storage pathology and preventing Purkinje cell death in a mouse model of Niemann-Pick A disease
    Passini, MA
    Bu, J
    Dodge, JC
    Zhao, Q
    Taksir, TV
    Collins, HA
    O'Riordan, CR
    Stewart, GR
    MOLECULAR THERAPY, 2004, 9 : S81 - S81
  • [30] Endostatin gene therapy on murine lung metastases model utilizing cationic vector-mediated intravenous gene delivery
    Nakashima, Y
    Yano, M
    Kobayashi, Y
    Moriyama, S
    Sasaki, H
    Toyama, T
    Yamashita, H
    Fukai, I
    Iwase, H
    Yamakawa, Y
    Fujii, Y
    GENE THERAPY, 2003, 10 (02) : 123 - 130
12345