QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A  G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

van Diepen Hester; Chan Heelam; Turunen Janne; Semo Maayan; Vugler Anthony; Slijkerman Ralph; van Wijk Erwin; Adamson Peter

首页> 外文期刊>Investigative ophthalmology & visual science >QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

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QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

机译：QRX-411，反义寡核苷酸（AON）引导USH2A mRNA中的接头矫正，由频繁的深入内肠道C.7595-2144A＆GT引起; G突变与母综合征2型中的视网膜炎

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来源
《Investigative ophthalmology & visual science》 |2017年第8期|共3页
作者
van Diepen Hester; Chan Heelam; Turunen Janne; Semo Maayan; Vugler Anthony; Slijkerman Ralph; van Wijk Erwin; Adamson Peter;
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作者单位

ProQR Therapeut Leiden Netherlands;

ProQR Therapeut Leiden Netherlands;

ProQR Therapeut Leiden Netherlands;

UCL Inst Ophthalmol London England;

UCL Inst Ophthalmol London England;

Radboud Univ Nijmegen Radboudumc Dept Otorhinolaryngol Nijmegen Netherlands;

Radboud Univ Nijmegen Radboudumc Dept Otorhinolaryngol Nijmegen Netherlands;

ProQR Therapeut Leiden Netherlands;

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正文语种 eng
中图分类眼科学;
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1. QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2 [J] . van Diepen Hester, Chan Heelam, Turunen Janne, Investigative ophthalmology & visual science . 2017,第8期

机译：QRX-411，反义寡核苷酸（AON）引导USH2A mRNA中的接头矫正，由频繁的深入内肠道C.7595-2144A＆GT引起; G突变与母综合征2型中的视网膜炎
2. QRX-421, an Antisense Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A, Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient Derived Optic Cups [J] . Adamson Peter, Chan Heelam, Turunen Janne, Investigative ophthalmology & visual science . 2017,第8期

机译：QRX-421，USH2A的外显子13中的反义寡核苷酸（AON）靶向突变，与映综合征2型中的视网膜炎，在患者成纤维细胞和患者衍生的视镜杯中的USH2A mRNA中有效
3. QRX-421, an Antisense Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A, Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient Derived Optic Cups [J] . Adamson Peter, Chan Heelam, Turunen Janne, Investigative ophthalmology & visual science . 2017,第8期

机译：QRX-421，USH2A的外显子13中的反义寡核苷酸（AON）靶向突变，与映综合征2型中的视网膜炎，在患者成纤维细胞和患者衍生的视镜杯中的USH2A mRNA中有效
4. Novel mutations in the long isoform of the USH2A gene in patients with Usher syndrome type II or non-syndromic retinitis pigmentosa [O] . Terri L. McGee, Babak Jian Seyedahmadi, Meredith O. Sweeney, -1

机译：USH2A基因患者患者综合征II型或非综合征视网膜炎患者的长同同族的新型突变
5. 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

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QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

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