Liver-directed AAV gene therapy corrects disease symptoms in a murine model of lysosomal acid lipase deficiency

被引:0
|
作者
Lam, Patricia [1 ]
Zygmunt, Deborah [1 ]
Ashbrook, Anna [1 ]
Yan, Cong [2 ]
Du, Hong [2 ]
Martin, Paul T. [1 ]
机构
[1] Nationwide Childrens Hosp, Abigail Wexner Res Inst, Columbus, OH USA
[2] Indiana Univ Sch Med, Indianapolis, IN USA
关键词
D O I
10.1016/j.ymgme.2023.107930
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
191
引用
收藏
页数:1
相关论文
共 50 条
  • [1] Liver-Directed AAV Gene Therapy for Gaucher Disease
    Miranda, Carlos J.
    Canavese, Miriam
    Chisari, Elisa
    Pandya, Jalpa
    Cocita, Clement
    Portillo, Maria
    McIntosh, Jenny
    Kia, Azadeh
    Foley, Jonathan H.
    Dane, Allison
    Jeyakumar, Jey M.
    Sheridan, Rose
    Corbau, Romuald
    Nathwani, Amit C.
    [J]. BLOOD, 2019, 134
  • [2] Liver-directed gene therapy corrects Fabry disease in mice
    Jeyakumar, Jey
    Kia, Azadeh
    McIntosh, Jenny
    Verhoef, Daniel
    Kalcheva, Petya
    Hosseini, Paniz
    Sheridan, Rose
    Corbau, Romuald
    Nathwani, Amit
    [J]. MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S80 - S80
  • [3] AAV8-mediated liver-directed gene therapy corrects blood phenylalanine in murine PKU.
    Harding, CO
    Gillingham, MB
    Hamman, K
    Clark, H
    Koeberl, D
    [J]. MOLECULAR GENETICS AND METABOLISM, 2005, 84 (03) : 222 - 222
  • [4] Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler
    Jin, Xiu
    Su, Jing
    Zhao, Qinyu
    Li, Ruiting
    Xiao, Jianlu
    Zhong, Xiaomei
    Song, Li
    Liu, Yi
    She, Kaiqin
    Deng, Hongxin
    Wei, Yuquan
    Yang, Yang
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2022, 25 : 370 - 381
  • [5] Development of AAV Gene Replacement Therapy for Lysosomal Acid Lipase Deficiency
    Lam, Patricia
    Ashbrook, Anna
    Zygmunt, Deborah
    Yang, Cong
    Du, Hong
    Martin, Paul T.
    [J]. MOLECULAR THERAPY, 2021, 29 (04) : 240 - 240
  • [6] Liver-Directed Gene Therapy for Ornithine Aminotransferase Deficiency
    Boffa, Iolanda
    Polishchuk, Elena
    De Stefano, Lucia
    Dell' Aquila, Fabio
    Nusco, Edoardo
    Marrocco, Elena
    Audano, Matteo
    Silvia, Pedretti
    Caterino, Marianna
    Bellezza, Ilaria
    Ruoppolo, Margherita
    Mitro, Nico
    Cellini, Barbara
    Auricchio, Alberto
    Brunetti-Pierri, Nicola
    [J]. MOLECULAR THERAPY, 2023, 31 (04) : 322 - 322
  • [7] Liver-directed gene therapy for ornithine aminotransferase deficiency
    Boffa, Iolanda
    Polishchuk, Elena
    De Stefano, Lucia
    Dell'Aquila, Fabio
    Nusco, Edoardo
    Marrocco, Elena
    Audano, Matteo
    Pedretti, Silvia
    Caterino, Marianna
    Bellezza, Ilaria
    Ruoppolo, Margherita
    Mitro, Nico
    Cellini, Barbara
    Auricchio, Alberto
    Brunetti-Pierri, Nicola
    [J]. EMBO MOLECULAR MEDICINE, 2023, 15 (04)
  • [8] Liver-directed gene therapy for murine glycogen storage disease type Ib
    Kwon, Joon Hyun
    Lee, Young Mok
    Cho, Jun-Ho
    Kim, Goo-Young
    Anduaga, Javier
    Starost, Matthew F.
    Mansfield, Brian C.
    Chou, Janice Y.
    [J]. HUMAN MOLECULAR GENETICS, 2017, 26 (22) : 4395 - 4405
  • [9] Systematic Evaluation of AAV Vectors for Liver-directed Gene Transfer in Murine Models
    Wang, Lili
    Wang, Huan
    Bell, Peter
    McCarter, Robert J.
    He, Jianping
    Calcedo, Roberto
    Vandenberghe, Luk H.
    Morizono, Hiroki
    Batshaw, Mark L.
    Wilson, James M.
    [J]. MOLECULAR THERAPY, 2010, 18 (01) : 118 - 125
  • [10] Gene therapy for lysosomal acid lipase deficiency in the mouse model.
    Du, H
    Heur, M
    Mishra, J
    Brannock, J
    Witte, D
    Rethmeier, J
    Ameis, D
    Grabowski, G
    [J]. AMERICAN JOURNAL OF HUMAN GENETICS, 2001, 69 (04) : 675 - 675