QR-110 Treatment for Leber's Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA10 iPSC-Derived Optic Cups

Biasutto Patricia; Dulla Kalyan; Adamson Peter; Schulkens Iris; Schmidt Iris; Lane Amelia; Aguila Monica; Cheetham Michael E.

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QR-110 Treatment for Leber's Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA10 iPSC-Derived Optic Cups

机译：QR-110治疗Leber的先天性生物症类型10：恢复Cep290 mRNA水平和LCA10 IPSC衍生光学杯中的纤维化

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来源
《Investigative ophthalmology & visual science 》 |2017年第8期| 共3页
作者
Biasutto Patricia; Dulla Kalyan; Adamson Peter; Schulkens Iris; Schmidt Iris; Lane Amelia; Aguila Monica; Cheetham Michael E.;
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作者单位

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

ProQR Therapeut Ophthalmol Leiden Netherlands;

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正文语种 eng
中图分类眼科学 ;
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专利

1. QR-110 Treatment for Leber's Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA10 iPSC-Derived Optic Cups [J] . Biasutto Patricia, Dulla Kalyan, Adamson Peter, Investigative ophthalmology & visual science . 2017 ,第8期

机译：QR-110治疗Leber的先天性生物症类型10：恢复Cep290 mRNA水平和LCA10 IPSC衍生光学杯中的纤维化
2. QR-110, an RNA oligonucleotide (AON) directing skipping of the cryptic exon in p.Cys998X CEP290 associated with Leber's congenital amaurosis, shows increase in wild-type mRNA and an ability to reach the outer nuclear layer (ONL) in mice following intravitreal administration [J] . Biasutto Patricia, Chan Hee Lam, Boersma Hester, Investigative ophthalmology & visual science . 2016 ,第12期

机译：QR-110，一种RNA寡核苷酸（AON）在与Leber先天性生物疲劳相关的P.Cys998x Cep290中跳过隐秘外显子的跳跃，显示出野生型mRNA的增加和在玻璃体内术后小鼠中的外核层（ONL）的能力行政
3. Preclinical assessment of in vivo gene editing efficiency, specificity, and tolerability of EDIT-101, an investigational CRISPR treatment for Leber congenital amaurosis 10 (LCA10) [J] . Stefanidakis M., Maeder M., Bounoutas G., Human gene therapy . 2018 ,第12期

机译：临床临床评估，体内基因编辑效率，特异性和编辑101的耐受性，对Leber先天性阿颈病10的研究克隆治疗（LCA10）
4. The Leber Congenital Amaurosis CEP290 Cat Model: Working Towards a Cure. [D] . Minella, Andrea Louise. 2017

机译：Leber先天性无门诊CEP290猫模型：正在努力治愈。
5. Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655AG LCA10 Models [O] . Kalyan Dulla, Monica Aguila, Amelia Lane, 2018

机译：剪接调节寡核苷酸QR-110在人类c.2991 + 1655A GLCA10模型中恢复CEP290 mRNA和功能
6. Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models [O] . Kalyan Dulla, Monica Aguila, Amelia Lane, 2018

机译：接头调节寡核苷酸QR-110恢复CEP290 mRNA和在人C.2991 + 1655A> G LCA10型号中的功能

QR-110 Treatment for Leber's Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA10 iPSC-Derived Optic Cups

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