Gene Augmentation Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa Rescues Rod Function and Promotes Cone Survival Long-Term

被引：0
|
作者：
Occelli, Laurence M. [1 ]
Michalakis, Stylianos [2 ]
Schoen, Christian [2 ]
Biel, Martin [2 ]
Petersen-Jones, Simon M. [1 ]
机构：
[1] VMC Michigan State Univ, E Lansing, MI USA
[2] LMU, CIPSM Dept Pharm, Ctr Drug Res, Munich, Germany
来源：
MOLECULAR THERAPY | 2018年 / 26卷 / 05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：
367
引用
收藏
页码：171 / 172
页数：2
相关论文
共 23 条
  • [1] Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa
    Mowat, Freya M.
    Occelli, Laurence M.
    Bartoe, Joshua T.
    Gervais, Kristen J.
    Bruewer, Ashlee R.
    Querubin, Janice
    Dinculescu, Astra
    Boye, Sanford L.
    Hauswirth, William W.
    Petersen-Jones, Simon M.
    FRONTIERS IN NEUROSCIENCE, 2017, 11
  • [2] Gene augmentation therapy in a large animal model of PDE6A-Retinitis Pigmentosa results in functional and structural rescue for at least 2.5 years
    Occelli, Laurence Mireille
    Michalakis, Stylianos
    Biel, Martin
    Petersen-Jones, Simon M.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2020, 61 (07)
  • [3] Gene Supplementation Rescues Rod Function and Preserves Photoreceptor and Retinal Morphology in Dogs, Leading the Way Toward Treating Human PDE6A-Retinitis Pigmentosa
    Occelli, Laurence M.
    Schoen, Christian
    Seeliger, Mathias W.
    Biel, Martin
    Michalakis, Stylianos
    Petersen-Jones, Simon M.
    HUMAN GENE THERAPY, 2017, 28 (12) : 1189 - 1201
  • [4] Cone function is dependent on rod rescue in a mouse model of Retinitis Pigmentosa gene therapy
    Scalabrino, Miranda
    Thapa, Mishek
    Zhang, Esther
    Peters, Lucy
    Pluenneke, Molly
    Nolt, Jay
    Chew, Lindsey
    Sampath, Alapakkam P.
    Chen, Jeannie
    Field, Greg
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
  • [5] Gene augmentation therapy in a large animal model of CNGB1 retinitis pigmentosa
    Petersen-Jones, Simon M.
    Occelli, Laurence Mireille
    Winkler, Paige
    Chiodo, Vince Chiodo
    Boye, Sanford L.
    Hauswirth, William W.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
  • [6] Activated mTORC1 promotes long-term cone survival in retinitis pigmentosa mice
    Venkatesh, Aditya
    Ma, Shan
    Le, Yun Z.
    Hall, Michael N.
    Rueegg, Markus A.
    Punzo, Claudio
    JOURNAL OF CLINICAL INVESTIGATION, 2015, 125 (04): : 1446 - 1458
  • [7] N-Acetylcysteine Promotes Long-Term Survival of Cones in a Model of Retinitis Pigmentosa
    Lee, Sun Young
    Usui, Shinichi
    Zafar, Abu-Bakr
    Oveson, Brian C.
    Jo, Young-Joon
    Lu, Lili
    Masoudi, Solmaz
    Campochiaro, Peter A.
    JOURNAL OF CELLULAR PHYSIOLOGY, 2011, 226 (07) : 1843 - 1849
  • [8] Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa
    Wert, Katherine J.
    Davis, Richard J.
    Sancho-Pelluz, Javier
    Nishina, Patsy M.
    Tsang, Stephen H.
    HUMAN MOLECULAR GENETICS, 2013, 22 (03) : 558 - 567
  • [9] Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
    Jiang, Li
    Zhang, Houbin
    Dizhoor, Alexander M.
    Boye, Shannon E.
    Hauswirth, William W.
    Frederick, Jeanne M.
    Baehr, Wolfgang
    PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2011, 108 (45) : 18476 - 18481
  • [10] Long-term neuroretinal full-thickness transplants in a large animal model of severe retinitis pigmentosa
    Fredrik Ghosh
    Karl Engelsberg
    Robert V. English
    Robert M. Petters
    Graefe's Archive for Clinical and Experimental Ophthalmology, 2007, 245 : 835 - 846
123