CN107406852B - 用于莱伯氏先天性黑蒙的寡核苷酸疗法 - Google Patents

用于莱伯氏先天性黑蒙的寡核苷酸疗法 Download PDF

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CN107406852B
CN107406852B CN201680012486.8A CN201680012486A CN107406852B CN 107406852 B CN107406852 B CN 107406852B CN 201680012486 A CN201680012486 A CN 201680012486A CN 107406852 B CN107406852 B CN 107406852B
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oligonucleotide
seq
cep290
aav
cell
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CN107406852A (zh
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P·C·比亚苏托
H·L·陈
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Thea Laboratories Ltd
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201680012486.8A 2015-02-27 2016-02-26 用于莱伯氏先天性黑蒙的寡核苷酸疗法 Active CN107406852B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201503408A GB201503408D0 (en) 2015-02-27 2015-02-27 Oligonucleotides
GB1503408.5 2015-02-27
PCT/EP2016/054164 WO2016135334A1 (en) 2015-02-27 2016-02-26 Oligonucleotide therapy for leber congenital amaurosis

Publications (2)

Publication Number Publication Date
CN107406852A CN107406852A (zh) 2017-11-28
CN107406852B true CN107406852B (zh) 2021-06-11

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Country Link
US (3) US10421963B2 (https=)
EP (1) EP3262170A1 (https=)
JP (1) JP6813506B2 (https=)
KR (1) KR102368918B1 (https=)
CN (1) CN107406852B (https=)
AU (1) AU2016223336B2 (https=)
CA (1) CA2976601C (https=)
EA (1) EA036791B1 (https=)
GB (1) GB201503408D0 (https=)
IL (1) IL253882B (https=)
MX (1) MX384479B (https=)
NZ (1) NZ735342A (https=)
WO (1) WO2016135334A1 (https=)
ZA (1) ZA201705331B (https=)

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GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
EP3840758A1 (en) 2018-08-21 2021-06-30 Deep Genomics Incorporated Therapeutic splice-switching oligonucleotides
JP2022518808A (ja) 2019-01-28 2022-03-16 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ レーバー先天黒内障の処置のためのアンチセンスオリゴヌクレオチド
US12398393B2 (en) 2019-01-28 2025-08-26 Proqr Therapeutics Ii B.V. RNA-editing oligonucleotides for the treatment of usher syndrome
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
CA3136172A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
JP7682808B2 (ja) 2019-04-26 2025-05-26 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル エクソンスキッピング戦略による網膜ジストロフィーの処置方法
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
CA3158528A1 (en) 2019-12-23 2021-07-01 Jim SWILDENS Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
EP4301856A2 (en) 2021-03-05 2024-01-10 ProQR Therapeutics II B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
CN113736812A (zh) * 2021-09-06 2021-12-03 武汉翼康基因科技有限公司 一种pcMINI载体及其构建方法与应用
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

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WO2012168435A1 (en) * 2011-06-10 2012-12-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of leber congenital amaurosis
WO2013036105A1 (en) * 2011-09-05 2013-03-14 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis

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WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2012168435A1 (en) * 2011-06-10 2012-12-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of leber congenital amaurosis
WO2013036105A1 (en) * 2011-09-05 2013-03-14 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis

Also Published As

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US10889817B2 (en) 2021-01-12
US20210139905A1 (en) 2021-05-13
JP2018507711A (ja) 2018-03-22
US20200063134A1 (en) 2020-02-27
US20180030439A1 (en) 2018-02-01
AU2016223336B2 (en) 2021-12-09
KR102368918B1 (ko) 2022-02-28
IL253882A0 (en) 2017-10-31
EA036791B1 (ru) 2020-12-22
EA201791820A1 (ru) 2017-12-29
EP3262170A1 (en) 2018-01-03
MX2017011013A (es) 2018-03-06
CA2976601A1 (en) 2016-09-01
US10421963B2 (en) 2019-09-24
JP6813506B2 (ja) 2021-01-13
MX384479B (es) 2025-03-14
US11920132B2 (en) 2024-03-05
IL253882B (en) 2022-03-01
ZA201705331B (en) 2019-07-31
NZ735342A (en) 2022-07-01
KR20170118755A (ko) 2017-10-25
BR112017018335A2 (pt) 2018-04-17
WO2016135334A1 (en) 2016-09-01
GB201503408D0 (en) 2015-04-15
CA2976601C (en) 2023-03-21
CN107406852A (zh) 2017-11-28
AU2016223336A1 (en) 2017-09-28

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