WO2019066549A3 - 망막 기능장애 질환 치료를 위한 유전자 조작 - Google Patents

망막 기능장애 질환 치료를 위한 유전자 조작 Download PDF

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WO2019066549A3
WO2019066549A3 PCT/KR2018/011522 KR2018011522W WO2019066549A3 WO 2019066549 A3 WO2019066549 A3 WO 2019066549A3 KR 2018011522 W KR2018011522 W KR 2018011522W WO 2019066549 A3 WO2019066549 A3 WO 2019066549A3
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treatment
retinal dysfunction
gene manipulation
gene
dysfunction disorder
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PCT/KR2018/011522
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English (en)
French (fr)
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WO2019066549A2 (ko
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이정민
송동우
김운기
김정훈
조동현
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주식회사 툴젠
서울대학교 산학협력단
서울대학교 병원
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Priority to EP18862381.3A priority Critical patent/EP3690045A2/en
Priority to US16/650,993 priority patent/US11845951B2/en
Publication of WO2019066549A2 publication Critical patent/WO2019066549A2/ko
Publication of WO2019066549A3 publication Critical patent/WO2019066549A3/ko

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Abstract

본 발명은 망막 기능장애 질환의 치료 또는 개선을 위한 유전자 조작용 조성물 또는 이를 이용한 방법에 관한 것이다. 보다 구체적으로, 망막 기능형성 유전자를 표적화할 수 있는 가이드핵산을 포함하는 유전자 조작용 조성물 및 이를 이용한 망막 기능형성 유전자를 인위적으로 조작 및/또는 교정하여 망막 기능장애로 인한 질환을 치료 또는 개선하는 방법에 관한 것이다.
PCT/KR2018/011522 2017-09-29 2018-09-28 망막 기능장애 질환 치료를 위한 유전자 조작 WO2019066549A2 (ko)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP18862381.3A EP3690045A2 (en) 2017-09-29 2018-09-28 Gene manipulation for treatment of retinal dysfunction disorder
US16/650,993 US11845951B2 (en) 2017-09-29 2018-09-28 Gene manipulation for treatment of retinal dysfunction disorder

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US201762565838P 2017-09-29 2017-09-29
US62/565,838 2017-09-29

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WO2019066549A2 WO2019066549A2 (ko) 2019-04-04
WO2019066549A3 true WO2019066549A3 (ko) 2019-07-04

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US (1) US11845951B2 (ko)
EP (1) EP3690045A2 (ko)
KR (1) KR102662879B1 (ko)
WO (1) WO2019066549A2 (ko)

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Publication number Priority date Publication date Assignee Title
EP3996739A4 (en) * 2019-07-10 2024-03-20 Emendobio Inc. DIFFERENTIAL INACTIVATION OF A HETEROZYGOUS ALLELE OF RPE65
CN111876418B (zh) * 2020-07-17 2024-02-02 北京希诺因生物科技有限公司 先天性黑蒙模型犬的建立方法
CN112522292B (zh) * 2020-10-29 2023-05-02 南京启真基因工程有限公司 一种用于构建先天性黑蒙症克隆猪核供体细胞的CRISPR/Cas9系统及其应用
WO2024026478A1 (en) * 2022-07-29 2024-02-01 Beam Therapeutics Inc. Compositions and methods for treating a congenital eye disease
CN116515908B (zh) * 2023-04-03 2024-04-26 苏州启辰生物科技有限公司 Rpe65基因编辑模型犬的建立方法

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Publication number Publication date
US11845951B2 (en) 2023-12-19
US20200277630A1 (en) 2020-09-03
WO2019066549A2 (ko) 2019-04-04
KR102662879B1 (ko) 2024-05-02
EP3690045A2 (en) 2020-08-05
KR20190038407A (ko) 2019-04-08

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