Discovering A Novel AAV Variant for Efficient RPE Transfection via Intravitreal Injection for Dry AMD Gene Therapy

被引:0
|
作者
Singh, Sonali [1 ]
Visel, Meike [2 ]
Byrne, Leah [2 ,3 ]
Ozturk, Bilge Esin [4 ]
Rickman, Catherine Bowes [5 ]
Flannery, John Gerard [1 ]
机构
[1] Univ Calif Berkeley, Vis Sci, Berkeley, CA 94720 USA
[2] Univ Calif Berkeley, Helen Wills Dept Neurosci, Berkeley, CA 94720 USA
[3] Univ Pittsburgh, Dept Ophthalmol, Pittsburgh, PA 15260 USA
[4] MeiraGTx HIdgs, London, England
[5] Duke Univ, Dept Ophthalmol, Durham, NC USA
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
5328
引用
收藏
页数:3
相关论文
共 50 条
  • [1] A novel AAV Vector for gene therapy of RPE-related retinal degenerative diseases via intravitreal delivery
    Gong, Yajun
    Huang, Xianyu
    Tu, Tianxiang
    Chu, Cenfeng
    Xian, Chunrui
    Yuan, Yushun
    Fu, Xin
    Li, Ruobi
    Zhong, Guisheng
    Zhou, Xiaolai
    MOLECULAR NEURODEGENERATION, 2024, 19 (01)
  • [2] Novel AAV capsid variant for efficient and effective inner ear gene therapy
    Rossi, A.
    Marx, J.
    Huang, P.
    Warnecke, A.
    Schambach, A.
    Stacker, H.
    Buening, H.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A31 - A31
  • [3] Novel AAV Vector for Delivery of Transgenes to the Photoreceptors and RPE via Intravitreal Injection Enables Rescue of Multiple Models of Retinal Disease
    Kumar-Singh, Rajendra
    Mishra, Manish
    MOLECULAR THERAPY, 2020, 28 (04) : 400 - 400
  • [4] Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
    Pavlou, Marina
    Schon, Christian
    Occelli, Laurence M.
    Rossi, Axel
    Meumann, Nadja
    Boyd, Ryan F.
    Bartoe, Joshua T.
    Siedlecki, Jakob
    Gerhardt, Maximilian J.
    Babutzka, Sabrina
    Bogedein, Jacqueline
    Wagner, Johanna E.
    Priglinger, Siegfried G.
    Biel, Martin
    Petersen-Jones, Simon M.
    Buening, Hildegard
    Michalakis, Stylianos
    EMBO MOLECULAR MEDICINE, 2021, 13 (04)
  • [5] A Penetrable AAV2 Capsid Variant for Efficient Intravitreal Gene Delivery to the Retina
    He, Xiaoyu
    Fu, Yidian
    Xu, Yangfan
    Ma, Liang
    Chai, Peiwei
    Shi, Hanhan
    Yao, Yizheng
    Ge, Shengfang
    Jia, Renbing
    Wen, Xuyang
    Yang, Zhi
    Fan, Xianqun
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2025, 66 (01)
  • [6] A novel dual AAV approach for intravitreal gene therapy of Stargardt disease
    Mehlfeld, V.
    Splith, V.
    Bartoschek, M.
    Pandurangi, J.
    Boehm, S.
    Riedmayr, L. M.
    Michalakis, S.
    Biel, M.
    Becirovic, E.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A26 - A26
  • [7] RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models
    Millington-Ward, Sophia
    Chadderton, Naomi
    Finnegan, Laura K.
    Post, Iris J. M.
    Carrigan, Matthew
    Nixon, Rachel
    Humphries, Marian M.
    Humphries, Pete
    Kenna, Paul F.
    Palfi, Arpad
    Farrar, G. Jane
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023, 24 (04)
  • [8] AAV Vector for Delivery of Transgenes to the Photoreceptors and RPE via Intravitreal Injection or cornea via Intracameral Injection Enables Rescue of Multiple Models of Ocular Disease
    Kumar-Singh, Rajendra
    Sivalingam, Kalaiselvi
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
  • [9] Efficiency of gene therapy via intravitreal injection in primate cones
    Giarmarco, Michelle M.
    Barborek, Rachel
    Rowlan, Jessica
    Rezeanu, Dragos
    Bembry, Briyana
    Neitz, Jay
    Neitz, Maureen
    Kuchenbecker, James A.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
  • [10] Novel AAV capsid variant for muscle-directed gene therapy
    Lemoine, J.
    Just, S.
    Dominguez, N.
    Galy, A.
    Buening, N.
    Richard, I.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A29 - A29