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
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
  • [1] GENE THERAPY IN A SHEEP MODEL OF CNGA3 ACHROMATOPSIA
    Banin, Eyal
    Obolensky, Alexey
    Ejzenberg, Ayala
    Zelinger, Lina
    Ezra-Elia, Raaya
    Rosov, Alexander
    Averbukh, Edward
    Gootwine, Elisha
    Ofri, Ron
    Hauswirth, William W.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2014, 55 (13)
  • [2] Comprehensive variant spectrum of the CNGA3 gene in patients affected by achromatopsia
    Solaki, Maria
    Baumann, Britta
    Reuter, Peggy
    Andreasson, Sten
    Audo, Isabelle
    Ayuso, Carmen
    Balousha, Ghassan
    Benedicenti, Francesco
    Birch, David
    Bitoun, Pierre
    Blain, Delphine
    Bocquet, Beatrice
    Branham, Kari
    Catala-Mora, Jaume
    De Baere, Elfride
    Dollfus, Helene
    Falana, Mohammed
    Giorda, Roberto
    Golovleva, Irina
    Gottlob, Irene
    Heckenlively, John R.
    Jacobson, Samuel G.
    Jones, Kaylie
    Jaegle, Herbert
    Janecke, Andreas R.
    Kellner, Ulrich
    Liskova, Petra
    Lorenz, Birgit
    Martorell-Sampol, Loreto
    Messias, Andre
    Meunier, Isabelle
    Belga Ottoni Porto, Fernanda
    Papageorgiou, Eleni
    Plomp, Astrid S.
    de Ravel, Thomy J. L.
    Reiff, Charlotte M.
    Renner, Agnes B.
    Rosenberg, Thomas
    Rudolph, Guenther
    Salati, Roberto
    Sener, E. Cumhur
    Sieving, Paul A.
    Stanzial, Franco
    Traboulsi, Elias, I
    Tsang, Stephen H.
    Varsanyi, Balazs
    Weleber, Richard G.
    Zobor, Ditta
    Stingl, Katarina
    Wissinger, Bernd
    HUMAN MUTATION, 2022, 43 (07) : 832 - 858
  • [3] Successful delivery of rAAV8.CNGA3 in a patient with CNGA3 achromatopsia
    Fischer, M. Dominik
    Wilhelm, Barbara
    Michalakis, Stylianos
    Zobor, Ditta
    Kohl, Susanne
    Seeliger, Matthias
    Zrenner, Eberhart
    Ueffing, Marius
    Wissinger, Bernd
    Biel, Martin
    Bartz-Schmidt, Karl Ulrich
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
  • [4] Achromatopsia caused by novel missense mutations in the CNGA3 gene
    Xi-Teng Chen
    Hui Huang
    Yan-Hua Chen
    Li-Jie Dong
    Xiao-Rong Li
    Xiao-Min Zhang
    International Journal of Ophthalmology, 2015, (05) : 910 - 915
  • [5] Novel CNGA3 mutations in Chinese patients with achromatopsia
    Liang, Xiaofang
    Dong, Fangtian
    Li, Hui
    Li, Huajin
    Yang, Lizhu
    Sui, Ruifang
    BRITISH JOURNAL OF OPHTHALMOLOGY, 2015, 99 (04) : 571 - 576
  • [6] Achromatopsia caused by novel missense mutations in the CNGA3 gene
    Chen, Xi-Teng
    Huang, Hui
    Chen, Yan-Hua
    Dong, Li-Jie
    Li, Xiao-Rong
    Zhang, Xiao-Min
    INTERNATIONAL JOURNAL OF OPHTHALMOLOGY, 2015, 8 (05) : 910 - 915
  • [7] Genetics and Disease Expression in the CNGA3 Form of Achromatopsia Steps on the Path to Gene Therapy
    Zelinger, Lina
    Cideciyan, Artur V.
    Kohl, Susanne
    Schwartz, Sharon B.
    Rosenmann, Ada
    Eli, Dalia
    Sumaroka, Alexander
    Roman, Alejandro J.
    Luo, Xunda
    Brown, Cassondra
    Rosin, Boris
    Blumenfeld, Anat
    Wissinger, Bernd
    Jacobson, Samuel G.
    Banin, Eyal
    Sharon, Dror
    OPHTHALMOLOGY, 2015, 122 (05) : 997 - 1007
  • [8] RECOVERY OF VISUAL FUNCTION FOLLOWING GENE THERAPY IN A LARGE ANIMAL MODEL OF CNGA3 ACHROMATOPSIA
    Averbukh, Edward
    Ofri, Ron
    Gootwine, Elisha
    Ezra-Elia, Raaya
    Honig, Hen
    Rosov, Alexander
    Yamin, Esther
    Obolensky, Alexey
    Hauswirth, William
    Banin, Eyal
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2013, 54 (15)
  • [9] Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia
    Banin, Eyal
    Gootwine, Elisha
    Obolensky, Alexey
    Ezra-Elia, Raaya
    Ejzenberg, Ayala
    Zelinger, Lina
    Honig, Hen
    Rosov, Alexander
    Yamin, Esther
    Sharon, Dror
    Averbukh, Edward
    Hauswirth, William W.
    Ofri, Ron
    MOLECULAR THERAPY, 2015, 23 (09) : 1423 - 1433
  • [10] Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia
    Ross, M.
    Obolensky, A.
    Averbukh, E.
    Desrosiers, M.
    Ezra-Elia, R.
    Honig, H.
    Yamin, E.
    Rosov, A.
    Dvir, H.
    Gootwine, E.
    Banin, E.
    Dalkara, D.
    Ofri, R.
    GENE THERAPY, 2022, 29 (10-11) : 624 - 635