Effects of Subretinal AAV8 Gene Therapy on Microperimetry in CNGA3 Achromatopsia Patients

被引：0
|
作者：
Ochakovski, G. Alex [1 ]
Zhour, Ahmad [1 ]
Kuehlewein, Laura [1 ]
Kohl, Susanne [2 ]
Rindtorff, Andrea [3 ]
Bartz-Schmidt, Karl Ulrich [1 ]
Ueffing, Marius [2 ]
Zrenner, Eberhart [1 ,2 ]
Michalakis, Stylianos [4 ]
Biel, Martin [4 ]
Wissinger, Bernd [2 ]
Peters, Tobias [3 ]
Wilhelm, Barbara [3 ]
Fischer, M. Dominik [1 ,2 ]
机构：
[1] Univ Hosp Tuebingen, Univ Eye Hosp, Ctr Ophthalmol, Tubingen, Germany
[2] Univ Hosp Tuebingen, Ctr Ophthalmol, Inst Ophthalm Res, Tubingen, Germany
[3] Univ Hosp Tuebingen, STZ Eyetrial Ctr Ophthalmol, Tubingen, Germany
[4] Ludwig Maximilians Univ Munchen, Ctr Drug Res, Ctr Integrated Prot Sci Munich CiPSM, Dept Pharm, Munich, Germany
来源：
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2019年 / 60卷 / 09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：
2921
引用
收藏
页数：4
相关论文
共 50 条
  • [41] IMPACT OF PEDF GENE THERAPY DELIVERED BY AAV8 IN MURINE MODEL OF CHRONIC ALLERGIC INFLAMMATION
    Ferreira, D. P.
    CYTOTHERAPY, 2021, 23 (05) : S153 - S154
  • [42] The Clinical Phenotype of CNGA3-Related Achromatopsia: Pretreatment Characterization in Preparation of a Gene Replacement Therapy Trial
    Zobor, Ditta
    Werner, Annette
    Stanzial, Franco
    Benedicenti, Francesco
    Rudolph, Gunther
    Kellner, Ulrich
    Hamel, Christian
    Andreasson, Sten
    Zobor, Gergely
    Strasser, Torsten
    Wissinger, Bernd
    Kohl, Susanne
    Zrenner, Eberhart
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2017, 58 (02) : 821 - 832
  • [43] Gene Therapy Restores Missing Cone-Mediated Vision in the CNGA3-/- Mouse Model of Achromatopsia
    Michalakis, Stylianos
    Muehlfriedel, Regine
    Tanimoto, Naoyuki
    Krishnamoorthy, Vidhyasankar
    Koch, Susanne
    Fischer, M. Dominik
    Becirovic, Elvir
    Bai, Lin
    Huber, Gesine
    Beck, Susanne C.
    Fahl, Edda
    Buening, Hildegard
    Schmidt, Jennifer
    Zong, Xiangang
    Gollisch, Tim
    Biel, Martin
    Seeliger, Mathias W.
    RETINAL DEGENERATIVE DISEASES, 2012, 723 : 183 - 189
  • [44] An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy
    Dastgir, J.
    Rastogi, S.
    Philips, D.
    Wilson, C.
    Boulos, N.
    Hall, J.
    Jimenez, V.
    Gilmor, M.
    Falabella, P.
    Owusu, L.
    Fiscella, M.
    Liu, Y.
    Pakola, S.
    Danos, O.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S101 - S101
  • [45] Evaluation of an AAV8 Gene Therapy Approach in a Mouse Model of Maple Syrup Urine Disease
    Greig, Jenny A.
    Ashley, Scott N.
    Nordin, Jayme M. L.
    Smith, Melanie K.
    Draper, Christine
    Wilson, James M.
    MOLECULAR THERAPY, 2018, 26 (05) : 252 - 252
  • [46] AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar
    Greig, Jenny A.
    Nordin, Jayme M. L.
    Draper, Christine
    Bell, Peter
    Wilson, James M.
    HUMAN GENE THERAPY, 2018, 29 (07) : 763 - 770
  • [47] The Importance of Transgene Optimization in Correcting the Dyslipidemia in a Mouse Model of Familial Hypercholesterolemia by Gene Therapy with AAV8
    Kassim, Sadik H.
    Vandenberghe, Luk H.
    Ziao, Ru
    Marchadier, Dawn
    Bell, Peter
    Cromley, Debra
    Wilson, James M.
    Rader, Daniel J.
    MOLECULAR THERAPY, 2009, 17 : S69 - S69
  • [48] Gene Therapy for MPS VI Is Effective in Cats without Pre-Existing Immunity to AAV8
    Ferla, Rita
    O'Malley, Thomas
    Calcedo, Roberto
    O'Donnell, Patricia
    Wang, Ping
    Cotugno, Gabriella
    Claudiani, Pamela
    Wilson, James M.
    Haskins, Mark
    Auricchio, Alberto
    MOLECULAR THERAPY, 2013, 21 : S138 - S139
  • [49] Transmembrane S1 mutations in CNGA3 from achromatopsia 2 patients cause loss of function and impaired cellular trafficking of the cone CNG channel
    Patel, KA
    Bartoli, KM
    Fandino, RA
    Ngatchou, AN
    Woch, G
    Carey, J
    Tanaka, JC
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2005, 46 (07) : 2282 - 2290
  • [50] A Novel AAV8 Vector for Microdystrophin Gene Therapy of Duchenne Muscular Dystrophy: Preclinical Studies in the Mdx Mouse
    Kim, SunJung
    Buss, Nicholas
    Patel, Hiren
    Yang, Lin
    Elliott, Kirk
    Qiao, Chunping
    Liu, Ye
    Fiscella, Michele
    Danos, Olivier
    MOLECULAR THERAPY, 2021, 29 (04) : 297 - 298
12345