EA202091105A1 - Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1) - Google Patents

Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1)

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EA202091105A1
EA202091105A1 EA202091105A EA202091105A EA202091105A1 EA 202091105 A1 EA202091105 A1 EA 202091105A1 EA 202091105 A EA202091105 A EA 202091105A EA 202091105 A EA202091105 A EA 202091105A EA 202091105 A1 EA202091105 A1 EA 202091105A1
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Prior art keywords
treatment
methods
guanylate cyclase
guanilatcyclase
amavrosis
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EA202091105A
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English (en)
Inventor
Шэннон Элизабет Бойе
Уилльям У. Хаузвирт
Сэнфорд Леон Бойе
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Юниверсити Оф Флорида Рисерч Фаундейшн, Инк.
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Publication of EA202091105A1 publication Critical patent/EA202091105A1/ru

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Abstract

Описаны композиции вирусного вектора, содержащие полинуклеотидные последовательности, которые экспрессируют один или несколько биологически активных гуанилатциклазных белков млекопитающих. Описаны также способы их применения в предотвращении, лечении и/или облегчении по меньшей мере одного или нескольких симптомов заболеваний, расстройств, аномальных состояний или дисфункций, являющихся, по меньшей мере отчасти, результатом дефицита гуанилатциклазы in vivo. В конкретных воплощениях применение рекомбинантных адено-ассоциированных вирусных векторов (rAAV) для лечения или облегчения симптомов врожденного амавроза Лебера, также как и других состояний, вызванных отсутствием или уменьшением экспрессии функциональной специфичной в отношении сетчатки гуанилатциклазы-1 (retGC1).
EA202091105A 2010-04-23 2011-04-22 Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1) EA202091105A1 (ru)

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EA202091105A EA202091105A1 (ru) 2010-04-23 2011-04-22 Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1)
EA201291103A EA035893B1 (ru) 2010-04-23 2011-04-22 РЕКОМБИНАНТНЫЙ АДЕНОАССОЦИИРОВАННЫЙ ВИРУСНЫЙ ВЕКТОР (rAAV), КОДИРУЮЩИЙ БИОЛОГИЧЕСКИ АКТИВНУЮ, СПЕЦИФИЧНУЮ В ОТНОШЕНИИ СЕТЧАТКИ ГУАНИЛАТЦИКЛАЗУ ЧЕЛОВЕКА, СПОСОБНЫЙ ПРЕДОХРАНЯТЬ ИЛИ ВОССТАНАВЛИВАТЬ ОПОСРЕДОВАННУЮ КОЛБОЧКАМИ ФУНКЦИЮ ГЛАЗА МЛЕКОПИТАЮЩЕГО

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EA201291103A EA035893B1 (ru) 2010-04-23 2011-04-22 РЕКОМБИНАНТНЫЙ АДЕНОАССОЦИИРОВАННЫЙ ВИРУСНЫЙ ВЕКТОР (rAAV), КОДИРУЮЩИЙ БИОЛОГИЧЕСКИ АКТИВНУЮ, СПЕЦИФИЧНУЮ В ОТНОШЕНИИ СЕТЧАТКИ ГУАНИЛАТЦИКЛАЗУ ЧЕЛОВЕКА, СПОСОБНЫЙ ПРЕДОХРАНЯТЬ ИЛИ ВОССТАНАВЛИВАТЬ ОПОСРЕДОВАННУЮ КОЛБОЧКАМИ ФУНКЦИЮ ГЛАЗА МЛЕКОПИТАЮЩЕГО

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US (3) US9816108B2 (ru)
EP (3) EP2561067B1 (ru)
JP (4) JP5897549B2 (ru)
KR (3) KR101984145B1 (ru)
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AU (1) AU2011242527B2 (ru)
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EA (2) EA202091105A1 (ru)
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