Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123)

Biasutto Patricia; Adamson Peter S.; Dulla Kalyan; Murray Sue; Monia Brett; McCaleb Michael

首页> 外文期刊>Investigative ophthalmology & visual science >Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123)

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Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123)

机译：等位基因特异性倒塌人P23H洛越蛋白酶mRNA和预防人源化P23H杂皮蛋白酶敲击小鼠的视网膜变性，用玻璃体外介绍器反义寡核苷酸处理后（QR-1123）

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来源
《Investigative ophthalmology & visual science》 |2019年第9期|共3页
作者
Biasutto Patricia; Adamson Peter S.; Dulla Kalyan; Murray Sue; Monia Brett; McCaleb Michael;
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作者单位

ProQR Therapeut Leiden Netherlands;

ProQR Therapeut Leiden Netherlands;

ProQR Therapeut Leiden Netherlands;

Ionis Pharmaceut Carlsbad CA USA;

Ionis Pharmaceut Carlsbad CA USA;

Ionis Pharmaceut Carlsbad CA USA;

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原文格式 PDF
正文语种 eng
中图分类眼科学;
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1. Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123) [J] . Biasutto Patricia, Adamson Peter S., Dulla Kalyan, Investigative ophthalmology & visual science . 2019,第9期

机译：等位基因特异性倒塌人P23H洛越蛋白酶mRNA和预防人源化P23H杂皮蛋白酶敲击小鼠的视网膜变性，用玻璃体外介绍器反义寡核苷酸处理后（QR-1123）
2. In vivo allele-specific CRISPR/Cas9 gene editing in the rhodopsin P23H knockin mouse model [J] . Li Pingjuan, Kleinstiver Benjamin, Leon Mihoko, Investigative ophthalmology & visual science . 2017,第8期

机译：在洛越蛋白P23H敲门鼠模型中的体内等位基因特异性CRAP / CAS9基因编辑
3. In vivo allele-specific CRISPR/Cas9 gene editing in the rhodopsin P23H knockin mouse model [J] . Li Pingjuan, Kleinstiver Benjamin, Leon Mihoko, Investigative ophthalmology & visual science . 2017,第8期

机译：在洛越蛋白P23H敲门鼠模型中的体内等位基因特异性CRAP / CAS9基因编辑
4. LEDGF1-326 Decreases P23H and Wild Type Rhodopsin Aggregates and P23H Rhodopsin Mediated Cell Damage in Human Retinal Pigment Epithelial Cells [O] . Rinku Baid, Robert I. Scheinman, Toshimichi Shinohara, 2011

机译：在人视网膜色素上皮细胞LEDGF1-326跌幅p23H和野生型视紫红质骨料和p23H视紫红质介导的细胞损伤
5. LEDGF1-326 Decreases P23H and Wild Type Rhodopsin Aggregates and P23H Rhodopsin Mediated Cell Damage in Human Retinal Pigment Epithelial Cells [O] . Baid, Rinku, Scheinman, Robert I., Shinohara, Toshimichi, 2011

机译：LEDGF1-326减少人视网膜色素上皮细胞中的P23H和野生型视紫红质聚集体以及P23H视紫红质介导的细胞损伤

Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123)

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