AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness

被引：0
|
作者：
Hirsch, Matthew [1 ,2 ]
Gilger, Brian C. [3 ]
Llanga, Telmo [1 ]
Davis, Richard [1 ]
Kurtzberg, Joanne [4 ]
Samulski, Richard J. [2 ]
Miyadera, Keiko [5 ]
机构：
[1] Univ N Carolina, Ophthalmol, Chapel Hill, NC USA
[2] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC USA
[3] North Carolina State Univ, Raleigh, NC USA
[4] Duke Univ, Durham, NC USA
[5] Univ Penn, Philadelphia, PA 19104 USA
来源：
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2017年 / 58卷 / 08期
关键词：
D O I：
暂无
中图分类号：
R77 [眼科学];
学科分类号：
100212 ;
摘要：
4480
引用
收藏
页数：3
相关论文
共 50 条
  • [1] AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness
    Llanga, Telmo A.
    Miyadera, Keiko
    Conatser, Laura
    Gilger, Brian
    Kurtzberg, Joanne
    Samulski, Richard J.
    Hirsch, Matthew
    MOLECULAR THERAPY, 2019, 27 (04) : 441 - 442
  • [2] AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness
    Song, Liujiang
    Miyadera, Keiko
    Llanga, Telmo A.
    Conatser, Laura
    Carlin, Kendall
    O'Donnell, Patricia
    Bagel, Jessica
    Gilger, Brian
    Hirsch, Matthew
    MOLECULAR THERAPY, 2018, 26 (05) : 412 - 412
  • [3] AAV Gene Therapy for MPS1 Corneal Clouding
    Hirsch, Matthew
    Samulski, Richard
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2015, 56 (07)
  • [4] AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness
    Vance, Melisa A.
    Hirsch, Matthew
    Samulski, Richard Jude
    Kurtzberg, Joanne
    Goodrich, Laurie
    Telmo, Llanga
    BLOOD, 2015, 126 (23)
  • [5] AAV Gene Therapy for MPS1-associated Corneal Blindness
    Vance, Melisa
    Llanga, Telmo
    Bennett, Will
    Woodard, Kenton
    Murlidharan, Giridhar
    Chungfat, Neil
    Asokan, Aravind
    Gilger, Brian
    Kurtzberg, Joanne
    Samulski, R. Jude
    Hirsch, Matthew L.
    SCIENTIFIC REPORTS, 2016, 6
  • [6] AAV Gene Therapy for MPS1-associated Corneal Blindness
    Melisa Vance
    Telmo Llanga
    Will Bennett
    Kenton Woodard
    Giridhar Murlidharan
    Neil Chungfat
    Aravind Asokan
    Brian Gilger
    Joanne Kurtzberg
    R. Jude Samulski
    Matthew L. Hirsch
    Scientific Reports, 6
  • [7] Intracorneal AAV Gene Therapy Prevents and Reverses Corneal Clouding in a Mucopolysaccharidosis I Canine Model - Translational Lessons and Promises
    Miyadera, Keiko
    Spector, Courtney
    Conatser, Laura
    Llanga, Telmo
    Gilger, Brian C.
    Hirsch, Matthew
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)
  • [8] Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I
    Miyadera, Keiko
    Conatser, Laura
    Llanga, Telmo A.
    Carlin, Kendall
    O'Donnell, Patricia
    Bagel, Jessica
    Song, Liujiang
    Kurtzberg, Joanne
    Samulski, R. Jude
    Gilger, Brian
    Hirsch, Matthew L.
    MOLECULAR THERAPY, 2020, 28 (06) : 1455 - 1463
  • [9] AAV Gene Therapy Prevents and Reverses Heart Failure in a Murine Knockout Model of Barth Syndrome
    Wang, Suya
    Li, Yifei
    Xu, Yang
    Ma, Qing
    Lin, Zhiqiang
    Schlame, Michael
    Bezzerides, Vassilios J.
    Strathdee, Douglas
    Pu, William T.
    CIRCULATION RESEARCH, 2020, 126 (08) : 1024 - 1039
  • [10] Results of gene therapy in children with Hurler syndrome (MPS1)
    Will, A
    Lashford, L
    Wraith, J
    Wynn, R
    Spooncer, E
    Hatton, C
    Besley, G
    Cooper, A
    Whitehead, K
    Anson, D
    Hopwood, I
    Dexter, TM
    Fairbairn, L
    BONE MARROW TRANSPLANTATION, 1999, 23 : S87 - S87
12345