RU2011149094A - Генная терапия нейродегенеративных нарушений - Google Patents
Генная терапия нейродегенеративных нарушений Download PDFInfo
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Abstract
1. Самокомплементарный аденоассоциированный вирусный (scAAV) вектор, который содержит полинуклеотид, кодирующий белок, который модулирует двигательную функцию у субъекта с нарушением двигательных нейронов.2. Вектор scAAV по п.1, где нарушение двигательных нейронов выбрано из спинальной мышечной атрофии (SMA), бокового амиотрофического склероза (ALS), спинобульбарной мышечной атрофии, спиноцеребеллярной атаксии, первичного латерального склероза (PLS) или травматического повреждения спинного мозга.3. Вектор scAAV по п.2, где нарушение двигательных нейронов представляет собой SMA.4. Вектор scAAV по п.3, где полинуклеотид кодирует белок выживаемости двигательных нейронов (SMN).5. Вектор scAAV по п.4, где белок SMN кодируется SMN-1 человека.6. Вектор scAAV по п.5, где белок SMN содержит аминокислотную последовательность, обладающую по меньшей мере 90% идентичностью последовательности с последовательностью, изображенной на фиг.9B.7. Вектор scAAV по п.6, где белок SMN содержит аминокислотную последовательность, как изображено на фиг.9B.8. Рекомбинантный вирион AAV, содержащий вектор scAAV по любому из пп.1-7.9. Композиция, содержащая рекомбинантный вирион AAV по п.8 и фармацевтически приемлемый эксципиент.10. Способ модуляции двигательной функции у субъекта с нарушением двигательных нейронов, включающий введение терапевтически эффективного количества композиции по п.9 в клетки субъекта.11. Способ обеспечения белка SMN у субъекта со спинальной мышечной атрофией (SMA), включающий введение рекомбинантного вириона AAV, содержащего вектор AAV по любому из пп.4-7, в клетки субъекта, нуждающегося в этом.12. Способ по любому из пп.10 или 11, в котором композицию вводят посредством введени
Claims (19)
1. Самокомплементарный аденоассоциированный вирусный (scAAV) вектор, который содержит полинуклеотид, кодирующий белок, который модулирует двигательную функцию у субъекта с нарушением двигательных нейронов.
2. Вектор scAAV по п.1, где нарушение двигательных нейронов выбрано из спинальной мышечной атрофии (SMA), бокового амиотрофического склероза (ALS), спинобульбарной мышечной атрофии, спиноцеребеллярной атаксии, первичного латерального склероза (PLS) или травматического повреждения спинного мозга.
3. Вектор scAAV по п.2, где нарушение двигательных нейронов представляет собой SMA.
4. Вектор scAAV по п.3, где полинуклеотид кодирует белок выживаемости двигательных нейронов (SMN).
5. Вектор scAAV по п.4, где белок SMN кодируется SMN-1 человека.
6. Вектор scAAV по п.5, где белок SMN содержит аминокислотную последовательность, обладающую по меньшей мере 90% идентичностью последовательности с последовательностью, изображенной на фиг.9B.
7. Вектор scAAV по п.6, где белок SMN содержит аминокислотную последовательность, как изображено на фиг.9B.
8. Рекомбинантный вирион AAV, содержащий вектор scAAV по любому из пп.1-7.
9. Композиция, содержащая рекомбинантный вирион AAV по п.8 и фармацевтически приемлемый эксципиент.
10. Способ модуляции двигательной функции у субъекта с нарушением двигательных нейронов, включающий введение терапевтически эффективного количества композиции по п.9 в клетки субъекта.
11. Способ обеспечения белка SMN у субъекта со спинальной мышечной атрофией (SMA), включающий введение рекомбинантного вириона AAV, содержащего вектор AAV по любому из пп.4-7, в клетки субъекта, нуждающегося в этом.
12. Способ по любому из пп.10 или 11, в котором композицию вводят посредством введения в по меньшей мере одну область глубоких ядер мозжечка.
13. Способ по любому из пп.10 или 11, в котором композицию вводят посредством прямой инъекции в спинной мозг.
14. Способ по любому из пп.10 или 11, в котором композицию вводят посредством интрацеребровентрикулярной инъекции.
15. Способ по п.14, в котором композицию вводят в по меньшей мере один боковой желудочек головного мозга.
16. Способ по любому из пп.10 или 11, в котором композицию вводят посредством как интрацеребровентрикулярной инъекции, так и прямой инъекции в спинной мозг.
17. Способ по любому из пп.10 или 11, в котором композицию вводят посредством интратекальной инъекции.
18. Применение рекомбинантного вириона AAV по п.8 в производстве лекарственного средства для модуляции двигательной функции у субъекта с нарушением двигательных нейронов.
19. Применение рекомбинантного вириона AAV, содержащего вектор AAV по любому из пп.4-7, в производстве лекарственного средства для обеспечения белка SMN в клетках субъекта со спинальной мышечной атрофией (SMA).
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US17498209P | 2009-05-02 | 2009-05-02 | |
US61/174,982 | 2009-05-02 | ||
US26805909P | 2009-06-08 | 2009-06-08 | |
US61/268,059 | 2009-06-08 | ||
PCT/US2010/001239 WO2010129021A1 (en) | 2009-05-02 | 2010-04-27 | Gene therapy for neurodegenerative disorders |
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US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
RU2603740C2 (ru) | 2009-05-02 | 2016-11-27 | Джензим Корпорейшн | Генная терапия нейродегенеративных нарушений |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
CA3066596A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
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