Antisense oligonucleotide-based correction of deep-intronic ABCA4 splice mutations using patient-derived fibroblasts and photoreceptor precursor cells

Naessens Sarah; Collin Rob W. J.; Coppieters Frauke; Bauwens Miriam; Duijkers Lonneke; Balikova Irina; Leroy Bart P.; De Baere Elfride; Garanto Alex

首页> 外文期刊>Investigative ophthalmology & visual science >Antisense oligonucleotide-based correction of deep-intronic ABCA4 splice mutations using patient-derived fibroblasts and photoreceptor precursor cells

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Antisense oligonucleotide-based correction of deep-intronic ABCA4 splice mutations using patient-derived fibroblasts and photoreceptor precursor cells

机译：使用患者衍生的成纤维细胞和光感受器前体细胞的深入内肠道ABCA4接头突变的反义寡核苷酸的校正

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来源
《Investigative ophthalmology & visual science》 |2019年第9期|共3页
作者
Naessens Sarah; Collin Rob W. J.; Coppieters Frauke; Bauwens Miriam; Duijkers Lonneke; Balikova Irina; Leroy Bart P.; De Baere Elfride; Garanto Alex;
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作者单位

Univ Ghent Ghent Belgium;

Radboud Univ Nijmegen Med Ctr Dept Human Genet Nijmegen Netherlands;

Univ Ghent Ghent Belgium;

Univ Ghent Ghent Belgium;

Radboud Univ Nijmegen Med Ctr Dept Human Genet Nijmegen Netherlands;

Univ Ghent Ghent Belgium;

Univ Ghent Ghent Belgium;

Univ Ghent Ghent Belgium;

Radboud Univ Nijmegen Med Ctr Dept Human Genet Nijmegen Netherlands;

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正文语种 eng
中图分类眼科学;
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专利

1. Antisense oligonucleotide-based correction of deep-intronic ABCA4 splice mutations using patient-derived fibroblasts and photoreceptor precursor cells [J] . Naessens Sarah, Collin Rob W. J., Coppieters Frauke, Investigative ophthalmology & visual science . 2019,第9期

机译：使用患者衍生的成纤维细胞和光感受器前体细胞的深入内肠道ABCA4接头突变的反义寡核苷酸的校正
2. Antisense oligonucleotide-based restoration of ABCA4 splicing defects caused by deep-intronic mutations associated with Stargardt disease [J] . Garanto Alex, Sangermano Riccardo, Albert Silvia, Investigative ophthalmology & visual science . 2018,第9期

机译：基于反义寡核苷酸的ABCA4剪接缺陷的恢复引起的与晕虫病相关的深入内肠突变
3. Antisense Oligonucleotide-Based Splice Correction of a Deep-Intronic Mutation in CHM Underlying Choroideremia [J] . Garanto Alejandro, Van der Velde-Visser Saskia D., Cremers Frans P. M., Advances in Experimental Medicine and Biology . 2018,第期

机译：基于反义的寡核苷酸的CHM底层血管内血症深入血清突变的剪切校正
4. Multimodal high-resolution imaging of photoreceptor precursor cells using gold nanoparticles [C] . Oshra Betzer, Yoav Chemla, Amos Markus, International Conference on Nanoscale Imaging, Sensing, and Actuation for Biomedical Applications . 2020

机译：使用金纳米粒子的光感受器前体细胞多模式高分辨率成像
5. Transforming potential of alternatively spliced variants of fibroblast growth factor receptor 2 in human mammary epithelial cells [D] . Moffa, Allison Beth 2006

机译：成纤维细胞生长因子受体2的可变剪接变体在人乳腺上皮细胞中的转化潜力
6. Deep-intronic ABCA4 variants explain missing heritability in Stargardt disease and allow correction of splice defects by antisense oligonucleotides [O] . Riccardo Sangermano, Alejandro Garanto, Mubeen Khan, -1

机译：深度内含子ABCA4变体解释了Stargardt病缺失的遗传力并允许通过反义寡核苷酸纠正剪接缺陷
7. Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation [O] . Radulfus WN Slijkerman, Christel Vaché, Margo Dona, 2016

机译：基于反义寡核苷酸的剪接修复因常见的深内含子突变引起的UsH2a相关性视网膜变性

Antisense oligonucleotide-based correction of deep-intronic ABCA4 splice mutations using patient-derived fibroblasts and photoreceptor precursor cells

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