Novel gene based therapies for inherited retinal dystrophies in pediatric patients

被引:0
|
作者
Stieger, K. [1 ]
机构
[1] Justus Liebig Univ Giessen, Dept Ophthalmol, Giessen, Germany
关键词
D O I
10.1111/j.1755-3768.2017.04414
中图分类号
R77 [眼科学];
学科分类号
100212 ;
摘要
引用
收藏
页数:1
相关论文
共 50 条
  • [21] New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies
    Pulman, Juliette
    Sahel, Jose-Alain
    Dalkara, Deniz
    CRISPR JOURNAL, 2022, 5 (03): : 377 - 388
  • [22] Gene supplementation therapy for recessive forms of inherited retinal dystrophies
    A J Smith
    J W B Bainbridge
    R R Ali
    Gene Therapy, 2012, 19 : 154 - 161
  • [23] A new approach based on targeted pooled DNA sequencing identifies novel mutations in patients with Inherited Retinal Dystrophies
    Maitane Ezquerra-Inchausti
    Ander Anasagasti
    Olatz Barandika
    Gonzaga Garay-Aramburu
    Marta Galdós
    Adolfo López de Munain
    Cristina Irigoyen
    Javier Ruiz-Ederra
    Scientific Reports, 8
  • [24] A new approach based on targeted pooled DNA sequencing identifies novel mutations in patients with Inherited Retinal Dystrophies
    Ezquerra-Inchausti, Maitane
    Anasagasti, Ander
    Barandika, Olatz
    Garai-Aramburu, Gonzaga
    Galdos, Marta
    Lopez de Munain, Adolfo
    Irigoyen, Cristina
    Ruiz-Ederra, Javier
    SCIENTIFIC REPORTS, 2018, 8
  • [25] Gene and cell-based therapies for inherited retinal disorders: An update
    Sengillo, Jesse D.
    Justus, Sally
    Tsai, Yi-Ting
    Cabral, Thiago
    Tsang, Stephen H.
    AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS, 2016, 172 (04) : 349 - 366
  • [26] Descriptive Study of a Cohort of 488 Patients with Inherited Retinal Dystrophies
    Coco-Martin, Rosa M.
    Diego-Alonso, Miguel
    Andres Orduz-Montana, W.
    Rosa Sanabria, M.
    Sanchez-Tocin, Hortensia
    CLINICAL OPHTHALMOLOGY, 2021, 15 : 1075 - 1084
  • [27] Gene editing in human iPSC models derived from patients affected by inherited retinal dystrophies
    Siles, Laura
    Navines-Ferrer, Arnau
    Ruiz-Nogales, Sheila
    Pomares, Esther
    EUROPEAN JOURNAL OF HUMAN GENETICS, 2023, 31 : 638 - 638
  • [28] Management and treatment of inherited retinal dystrophies
    Levi, Sarah R.
    Jenny, Laura A.
    Tsang, Stephen H.
    TAIWAN JOURNAL OF OPHTHALMOLOGY, 2021, 11 (03) : 205 - 206
  • [29] Zebrafish models of inherited retinal dystrophies
    Perkins, Brian D.
    JOURNAL OF TRANSLATIONAL GENETICS AND GENOMICS, 2022, 6 (01) : 95 - 110
  • [30] Intraretinal pigment migration predicts gene location in inherited retinal dystrophies
    Levi, Sarah
    Oh, Jin Kyun
    Kim, Joonpyo
    Lima Carvalho-, Jose Ronaldo, Jr.
    Ryu, Joseph
    Tsang, Stephen
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2020, 61 (07)