CN111867635A - 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 - Google Patents

包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 Download PDF

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CN111867635A
CN111867635A CN201880081245.8A CN201880081245A CN111867635A CN 111867635 A CN111867635 A CN 111867635A CN 201880081245 A CN201880081245 A CN 201880081245A CN 111867635 A CN111867635 A CN 111867635A
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gly
polynucleotide
ala
seq
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丹妮兹·达尔卡拉
哈南·卡布
若泽-阿兰·萨赫勒
蒂埃里·莱维拉德
延斯·迪贝尔
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French National Association Of Health And Research Medicine
Sorbonne Universite
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Sorbonne Universite
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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CN201880081245.8A 2017-10-20 2018-10-22 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 Pending CN111867635A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17306430.4 2017-10-20
EP17306430 2017-10-20
PCT/EP2018/078856 WO2019077159A1 (en) 2017-10-20 2018-10-22 METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR

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CN111867635A true CN111867635A (zh) 2020-10-30

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US (1) US11723988B2 (https=)
EP (1) EP3697448A1 (https=)
JP (1) JP7420710B2 (https=)
KR (1) KR102763312B1 (https=)
CN (1) CN111867635A (https=)
WO (1) WO2019077159A1 (https=)

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CN113121655A (zh) * 2021-04-19 2021-07-16 上海信致医药科技有限公司 眼部和肌肉特异靶向型腺相关病毒载体及其应用
CN117247434A (zh) * 2023-11-10 2023-12-19 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
CN119350454A (zh) * 2024-12-27 2025-01-24 上海朗昇生物科技有限公司 衣壳重排型腺相关病毒载体及其用途

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CN110709095B (zh) 2017-04-05 2024-05-03 马萨诸塞大学 小基因治疗
WO2020236815A1 (en) 2019-05-20 2020-11-26 University Of Massachusetts Minigene therapy
US20230159609A1 (en) * 2020-04-10 2023-05-25 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
CA3195696A1 (en) * 2020-09-17 2022-03-24 Restore Vision Inc. Composition for treating or preventing diseases, disorders, or conditions associated with endoplasmic reticulum stress or all-trans-retinal, protecting retinal thickness, or suppressing reduction in retinal thickness or progress of reduction in retinal thickness
WO2022223644A2 (en) 2021-04-20 2022-10-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for treating retinal degenerative disorders
CA3221439A1 (en) * 2021-05-26 2022-12-01 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
IL320351A (en) 2022-10-20 2025-06-01 Sparingvision Compositions and methods for treating retinal degenerative disorders
WO2024110770A1 (en) * 2022-11-22 2024-05-30 INSERM (Institut National de la Santé et de la Recherche Médicale) A new promoter for retinal pigment epithelium (rpe) targeted gene therapy
WO2024182558A1 (en) * 2023-02-28 2024-09-06 The Saban Research Institute; Children's Hospital Los Angeles Fluorescent cone reporter ips cells, retinal organoids and uses thereof

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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113121655A (zh) * 2021-04-19 2021-07-16 上海信致医药科技有限公司 眼部和肌肉特异靶向型腺相关病毒载体及其应用
CN117247434A (zh) * 2023-11-10 2023-12-19 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
CN117247434B (zh) * 2023-11-10 2024-02-02 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
WO2025098512A1 (zh) * 2023-11-10 2025-05-15 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
CN119350454A (zh) * 2024-12-27 2025-01-24 上海朗昇生物科技有限公司 衣壳重排型腺相关病毒载体及其用途

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KR20200103634A (ko) 2020-09-02
JP2021500030A (ja) 2021-01-07
KR102763312B1 (ko) 2025-02-07
US20210268125A1 (en) 2021-09-02
WO2019077159A1 (en) 2019-04-25
US11723988B2 (en) 2023-08-15
JP7420710B2 (ja) 2024-01-23
EP3697448A1 (en) 2020-08-26

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