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RU2013151885A - Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования - Google Patents

Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования Download PDF

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RU2013151885A
RU2013151885A RU2013151885/10A RU2013151885A RU2013151885A RU 2013151885 A RU2013151885 A RU 2013151885A RU 2013151885/10 A RU2013151885/10 A RU 2013151885/10A RU 2013151885 A RU2013151885 A RU 2013151885A RU 2013151885 A RU2013151885 A RU 2013151885A
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amino acids
seq
virion
capsid protein
cell
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RU2013151885/10A
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RU2611202C2 (ru
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Дэвид В. ШЕФФЕР
Райан Р. КЛИМЧАК
Джеймс Т. КЁРБЕР
Джон Дж. ФЛЭННЕРИ
МОУРОТ Дениз ДАЛКАРА
Майке ВИЗЕЛЬ
Леа С.Т. БЁРН
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Те Риджентс Оф Те Юниверсити Оф Калифорния
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Abstract

1. Вирион рекомбинантного вируса (rAAV), содержащий:a) вариантный капсидный белок AAV, отличающийся тем, что вариантный капсидный белок AAV содержит вставку из приблизительно 5 аминокислот до приблизительно 11 аминокислот в капсидном белке петли GH по сравнению с соответствующим родительским капсидным белком AAV, и где вариантный капсидный белок дает повышенную инфекционность ретинальной клетки по сравнению с инфекционностью ретинальной клетки вирионом AAV, содержащим соответствующий родительский капсидный белок AAV; иb) гетерологичную нуклеиновую кислоту, содержащую нуклеотидную последовательность, кодирующую генный продукт.2. Вирион rAAV по п.1, отличающийся тем, что вставка является пептидом Формулы I, Формулы IIa, Формулы III или Формулы IV.3. Вирион rAAV по п.2, отличающийся тем, что вставка содержит аминокислотную последовательность, выбранную из LGETTRP (SEQ ID №13), NETITRP (SEQ ID №14), KAGQANN (SEQ ID №15), KDPKTTN (SEQ ID №16), KDTDTTR (SEQ ID №57), RAGGSVG (SEQ ID №58), AVDTTKF (SEQ ID №59) и STGKVPN (SEQ ID №60).4. Вирион rAAV по п.1, отличающийся тем, что ретинальная клетка является фоторецептором, ганглиозной клеткой сетчатки, клеткой Мюллера, биполярной клеткой, амакринной клеткой, горизонтальной клеткой или клеткой или клеткой пигментного эпителия сетчатки.5. Вирион rAAV по п.1, отличающийся тем, что сайт встраивания находится между аминокислотами 587 и 588 из AAV2, между аминокислотами 590 и 591 из AAV1, между аминокислотами 575 и 576 из AAV5, между аминокислотами 590 и 591 из AAV6, между аминокислотами 589 и 590 из AAV7, между аминокислотами 590 и 591 из AAV8, между аминокислотами 588 и 589 из AAV9 или между аминокислотами 588 и 589 из AAV10.6. Вирион rAAV по п.1, отличающийся тем, что вирион rAAV демонстрирует, по меньшей мере 10-кратное пов�

Claims (27)

1. Вирион рекомбинантного вируса (rAAV), содержащий:
a) вариантный капсидный белок AAV, отличающийся тем, что вариантный капсидный белок AAV содержит вставку из приблизительно 5 аминокислот до приблизительно 11 аминокислот в капсидном белке петли GH по сравнению с соответствующим родительским капсидным белком AAV, и где вариантный капсидный белок дает повышенную инфекционность ретинальной клетки по сравнению с инфекционностью ретинальной клетки вирионом AAV, содержащим соответствующий родительский капсидный белок AAV; и
b) гетерологичную нуклеиновую кислоту, содержащую нуклеотидную последовательность, кодирующую генный продукт.
2. Вирион rAAV по п.1, отличающийся тем, что вставка является пептидом Формулы I, Формулы IIa, Формулы III или Формулы IV.
3. Вирион rAAV по п.2, отличающийся тем, что вставка содержит аминокислотную последовательность, выбранную из LGETTRP (SEQ ID №13), NETITRP (SEQ ID №14), KAGQANN (SEQ ID №15), KDPKTTN (SEQ ID №16), KDTDTTR (SEQ ID №57), RAGGSVG (SEQ ID №58), AVDTTKF (SEQ ID №59) и STGKVPN (SEQ ID №60).
4. Вирион rAAV по п.1, отличающийся тем, что ретинальная клетка является фоторецептором, ганглиозной клеткой сетчатки, клеткой Мюллера, биполярной клеткой, амакринной клеткой, горизонтальной клеткой или клеткой или клеткой пигментного эпителия сетчатки.
5. Вирион rAAV по п.1, отличающийся тем, что сайт встраивания находится между аминокислотами 587 и 588 из AAV2, между аминокислотами 590 и 591 из AAV1, между аминокислотами 575 и 576 из AAV5, между аминокислотами 590 и 591 из AAV6, между аминокислотами 589 и 590 из AAV7, между аминокислотами 590 и 591 из AAV8, между аминокислотами 588 и 589 из AAV9 или между аминокислотами 588 и 589 из AAV10.
6. Вирион rAAV по п.1, отличающийся тем, что вирион rAAV демонстрирует, по меньшей мере 10-кратное повышение инфекционности ретинальной клетки по сравнению с инфекционностью ретинальной клетки вирионом AAV, содержащим соответствующий родительский капсидный белок AAV.
7. Вирион rAAV по п.1, отличающийся тем, что вирион rAAV демонстрирует, по меньшей мере 50-кратное повышение инфекционности ретинальной клетки по сравнению с инфекционностью ретинальной клетки вирионом AAV, содержащим соответствующий родительский капсидный белок AAV.
8. Вирион rAAV по п.1, отличающийся тем, что генный продукт является интерферирующей РНК или аптамером.
9. Вирион rAAV по п.1, отличающийся тем, что генный продукт является полипептидом.
10. Вирион rAAV по п.7, отличающийся тем, что полипептид является нейропротекторным полипептидом, анти-ангиогенным полипептидом или полипептидом, который повышает функцию ретинальной клетки.
11. Фармацевтическая композиция, содержащая:
a) вирион рекомбинантного аденоассоциированного вируса по п.1; и
b) фармацевтически приемлемый эксципиент.
12. Способ доставки генного продукта в ретинальную клетку индивидуума, где способ включает введение индивидууму вириона рекомбинантного аденоассоциированного вируса (rAAV) по п.1.
13. Способ по п.12, отличающийся тем, что генный продукт является полипептидом.
14. Способ по п.12, отличающийся тем, что генный продукт является короткой интерферирующей РНК или аптамером.
15. Способ по п.13, отличающийся тем, что полипептид является нейропротекторным полипептидом, анти-ангиогенным полипептидом, анти-апоптическим фактором или полипептидом, который повышает функцию ретинальной клетки.
16. Способ по п.13, отличающийся тем, что полипептид является глиальным нейротрофическим фактором; фактором роста фибробластов 2; нейротурином; цилиарным нейротрофическим фактором; фактор роста нервов; нейротрофическим фактором из тканей мозга, эпидермальным фактором роста, родопсином, Х-сцепленным ингибитором апоптоза, ретиношизином, RPE65, взаимодействующим белком-1 регулятора ГТФазы пигментной дистрофии сетчатки, периферином, периферином-2, родопсином или хеджехог-белком.
17. Способ лечения заболевания сетчатки, который включает введение индивидууму, нуждающемуся в этом, эффективного количества вириона рекомбинантного аденоассоциированного вируса (rAAV) по п.1.
18. Способ по п.17, отличающийся тем, что указанное введение является внутриглазной инъекцией.
19. Способ по п.17, отличающийся тем, что указанное введение является интравитреальной инъекцией.
20. Способ по п.17, отличающийся тем, что глазная болезнь является глаукомой, пигментной дистрофией сетчатки, макулярной дегенерацией, ретиношизисом, врожденным амаврозом Лебера, диабетической ретинопатией, ахроматопсией или дальтонизмом.
21. Выделенная нуклеиновая кислота, содержащая нуклеотидную последовательность, которая кодирует вариантный капсидный белок аденоассоциированного вируса (AAV), отличающаяся тем, что вариантный капсидный белок AAV содержит вставку из приблизительно 5 аминокислот до приблизительно 11 аминокислот петле GH капсидного белка по сравнению с соответствующим родительским капсидным белком AAV, и где вариантный капсидный белок, когда присутствует в вирионе AAV обеспечивает повышенную инфекционность ретинальной клетки вирионом AAV.
22. Выделенная нуклеиновая кислота по п.21, отличающаяся тем, что сайт встраивания находится между аминокислотами 587 и 588 из AAV2, между аминокислотами 590 и 591 из AAV1, между аминокислотами 575 и 576 из AAV5, между аминокислотами 590 и 591 из AAV6, между аминокислотами 589 и 590 из AAV7, между аминокислотами 590 и 591 из AAV8, между аминокислотами 588 и 589 из AAV9 или между аминокислотами 588 и 589 из AAV10.
23. Выделенная генетически модифицированная клетка-хозяин, содержащая нуклеиновую кислоту по п.21.
24. Вариантный капсидный белок аденоассоциированного вируса (AAV), отличающийся тем, что вариантный капсидный белок AAV содержит вставку из приблизительно 5 аминокислот до приблизительно 11 аминокислот, причем аминокислотная вставка находится в петле GH нативного капсида AAV, где вставка является пептидом Формулы I, Формулы IIa, Формулы III или Формулы IV.
25. Вариантный капсидный белок AAV по п.24, отличающийся тем, что вставка содержит аминокислотную последовательность, выбранную из LGETTRP (SEQ ID №13), NETITRP (SEQ ID №14), KAGQANN (SEQ ID №15), KDPKTTN (SEQ ID №16), KDTDTTR (SEQ ID №57), RAGGSVG (SEQ ID №58), AVDTTKF (SEQ ID №59) и STGKVPN (SEQ ID №60).
26. Вариантный капсидный белок AAV по п.24, отличающийся тем, что сайт встраивания находится между аминокислотами 587 и 588 из AAV2, между аминокислотами 590 и 591 из AAV1, между аминокислотами 575 и 576 из AAV5, между аминокислотами 590 и 591 из AAV6, между аминокислотами 589 и 590 из AAV7, между аминокислотами 590 и 591 из AAV8, между аминокислотами 588 и 589 из AAV9 или между аминокислотами 588 и 589 из AAV10.
27. Нуклеиновая кислота, содержащая нуклеотидную последовательность, кодирующую вариантный капсидный белок AAV по п.24.
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