RU2020111038A - Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы - Google Patents

Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы Download PDF

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RU2020111038A
RU2020111038A RU2020111038A RU2020111038A RU2020111038A RU 2020111038 A RU2020111038 A RU 2020111038A RU 2020111038 A RU2020111038 A RU 2020111038A RU 2020111038 A RU2020111038 A RU 2020111038A RU 2020111038 A RU2020111038 A RU 2020111038A
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Питер ПЕЧАН
Абрахам СКАРИА
Джеффри АРДИНГЕР
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Джензим Корпорейшн
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Claims (19)

1. Способ доставки нуклеиновой кислоты в трабекулярную сеть глаза млекопитающего, включающий введение частицы AAV серотипа 2 (AAV2), содержащей вектор rAAV, в глаз млекопитающего, где вектор rAAV содержит нуклеиновую кислоту, и где частица AAV2 содержит капсидный белок AAV2, содержащий аминокислотную замену R471A, нумерация основана на VP1 AAV2.
2. Способ по п.1, в котором нуклеиновая кислота кодирует терапевтический трансген.
3. Способ лечения глазного расстройства у млекопитающего, включающий введение частицы AAV2, содержащей вектор rAAV, в глаз млекопитающего, где вектор rAAV содержит нуклеиновую кислоту, кодирующую терапевтический трансген, и где частица AAV2 содержит капсидный белок AAV2, содержащий аминокислотную замену R471A, нумерация основана на VP1 AAV2.
4. Способ по любому из пп. 1-3, в котором частица rAAV трансдуцирует клетки трабекулярной сети глаза.
5. Способ по любому из пп. 2-4, в котором терапевтический трансген экспрессируется в трабекулярной сети глаза.
6. Способ по любому из пп. 1-5, в котором терапевтический трансген кодирует терапевтический полипептид или терапевтическую нуклеиновую кислоту.
7. Способ по п.6, в котором глазное расстройство представляет собой расстройство, связанное с трабекулярной сеткой глаза.
8. Способ по п.7, в котором глазное расстройство представляет собой миоцилиновую (MYOC) глаукому.
9. Способ по любому из пп. 1-8, в котором млекопитающее является человеком.
10. Способ по любому из пп. 1-9, в котором введение частицы AAV является интравитреальным и/или интракамеральным.
11. Набор для доставки нуклеиновой кислоты в трабекулярную сеть глаза млекопитающего или для лечения глазного расстройства у млекопитающего, включающий частицу rAAV2, содержащую вектор rAAV, где вектор rAAV включает нуклеиновую кислоту, и где AAV2 частица содержит капсидный белок AAV2, содержащий аминокислотную замену R471A, нумерация основана на VP1 AAV2.
12. Набор по п.11, в котором нуклеиновая кислота кодирует терапевтический трансген.
13. Набор по п.11 или 12, в котором частица rAAV трансдуцирует клетки трабекулярной сети глаза.
14. Набор по п.12 или 13, где терапевтический трансген экспрессируется в трабекулярной сети глаза.
15. Набор по п.14, в котором глазное расстройство представляет собой расстройство, связанное с трабекулярной сеткой глаза.
16. Набор по п.14 или 15, в котором глазное расстройство представляет собой миоцилиновую (MYOC) глаукому.
17. Набор по любому из пп.11-16, в котором млекопитающее является человеком.
18. Набор по любому из пп. 11-17, дополнительно содержащий инструкции по применению.
19. Набор по любому из пп. 11-18, дополнительно содержащий буферы и/или фармацевтически приемлемые эксципиенты.
RU2020111038A 2014-09-16 2015-09-16 Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы RU2746991C2 (ru)

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EP3751000A4 (en) * 2018-02-07 2021-04-21 Nippon Medical School Foundation IMPROVED ADENO-ASSOCIATED VIRUS VECTOR
WO2019165241A1 (en) * 2018-02-22 2019-08-29 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
CA3131919A1 (en) 2019-02-28 2020-09-03 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
CN113032010B (zh) * 2021-03-12 2022-09-20 歌尔科技有限公司 命令的传输控制方法、终端及计算机可读存储介质
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WO2023114446A2 (en) * 2021-12-16 2023-06-22 Peter Koulen A machine learning-based framework using electroretinography for detecting early-stage glaucoma
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