RU2013116447A - Системы и способы доставки биоактивных средств с применением транспортных последовательностей, происходящих от бактериального токсина - Google Patents

Системы и способы доставки биоактивных средств с применением транспортных последовательностей, происходящих от бактериального токсина Download PDF

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RU2013116447A
RU2013116447A RU2013116447/10A RU2013116447A RU2013116447A RU 2013116447 A RU2013116447 A RU 2013116447A RU 2013116447/10 A RU2013116447/10 A RU 2013116447/10A RU 2013116447 A RU2013116447 A RU 2013116447A RU 2013116447 A RU2013116447 A RU 2013116447A
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delivery
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Рэндэлл Дж. МРСНИ
Тахир МАХМУД
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Рэндэлл Дж. МРСНИ
Тахир МАХМУД
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Abstract

1. Выделенный конструкт для доставки, содержащий транспортный домен и груз, причем указанный конструкт способен к доставке указанного груза в целевые внутриклеточные компартменты.2. Выделенный конструкт для доставки, содержащий транспортный домен и макромолекулу, причем указанный конструкт способен к доставке указанной макромолекулы к биологическому компартменту субъекта без инъекций.3. Конструкт по любому из п.1 или 2, дополнительно содержащий расщепляемый линкер.4. Конструкт по любому из п.1 или 2, где указанный транспортный домен включает полипептидную последовательность, происходящую от Cholix.5. Конструкт по п.1, где указанный груз выбран из группы, состоящей из макромолекул, малых молекул, siRNA, PNA, miRNA, ДНК, плазмиды и антисмысловых молекул.6. Применение конструкта по п.1 для доставки груза в целевые внутриклеточные компартменты.7. Применение слияния или конструкта по п.1 для доставки груза путем доставки наночастиц и носителей на основе дендримеров через биологические мембраны.8. Применение наночастиц или носителей на основе дендримеров по п.6, декорированных транспортным доменом, с или без линкеров для доставки груза через биологические мембраны.9. Фармацевтическая композиция, содержащая конструкт для доставки по любому из пп.1-5 и фармацевтически приемлемый носитель.10. Полинуклеотид, который кодирует конструкт для доставки, причем указанный конструкт для доставки содержит транспортный домен и груз, при этом указанный конструкт способен к доставке указанного груза в целевые внутриклеточные компартменты.11. Вектор экспрессии, содержащий полинуклеотид по п.10.12. Выделенная клетка-хозяин, содержащая вектор экспр�

Claims (12)

1. Выделенный конструкт для доставки, содержащий транспортный домен и груз, причем указанный конструкт способен к доставке указанного груза в целевые внутриклеточные компартменты.
2. Выделенный конструкт для доставки, содержащий транспортный домен и макромолекулу, причем указанный конструкт способен к доставке указанной макромолекулы к биологическому компартменту субъекта без инъекций.
3. Конструкт по любому из п.1 или 2, дополнительно содержащий расщепляемый линкер.
4. Конструкт по любому из п.1 или 2, где указанный транспортный домен включает полипептидную последовательность, происходящую от Cholix.
5. Конструкт по п.1, где указанный груз выбран из группы, состоящей из макромолекул, малых молекул, siRNA, PNA, miRNA, ДНК, плазмиды и антисмысловых молекул.
6. Применение конструкта по п.1 для доставки груза в целевые внутриклеточные компартменты.
7. Применение слияния или конструкта по п.1 для доставки груза путем доставки наночастиц и носителей на основе дендримеров через биологические мембраны.
8. Применение наночастиц или носителей на основе дендримеров по п.6, декорированных транспортным доменом, с или без линкеров для доставки груза через биологические мембраны.
9. Фармацевтическая композиция, содержащая конструкт для доставки по любому из пп.1-5 и фармацевтически приемлемый носитель.
10. Полинуклеотид, который кодирует конструкт для доставки, причем указанный конструкт для доставки содержит транспортный домен и груз, при этом указанный конструкт способен к доставке указанного груза в целевые внутриклеточные компартменты.
11. Вектор экспрессии, содержащий полинуклеотид по п.10.
12. Выделенная клетка-хозяин, содержащая вектор экспрессии по п.11.
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