RU2013151885A - Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования - Google Patents
Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования Download PDFInfo
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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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- Prior art keywords
- amino acids
- seq
- virion
- capsid protein
- cell
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- 241000702421 Dependoparvovirus Species 0.000 title claims abstract 11
- 238000000034 method Methods 0.000 title claims 11
- 150000001413 amino acids Chemical class 0.000 claims abstract 43
- 210000002845 virion Anatomy 0.000 claims abstract 27
- 108090000565 Capsid Proteins Proteins 0.000 claims abstract 24
- 102100023321 Ceruloplasmin Human genes 0.000 claims abstract 24
- 210000004027 cell Anatomy 0.000 claims abstract 17
- 108090000765 processed proteins & peptides Proteins 0.000 claims abstract 14
- 230000002207 retinal effect Effects 0.000 claims abstract 14
- 108090000623 proteins and genes Proteins 0.000 claims abstract 7
- 108020004707 nucleic acids Proteins 0.000 claims abstract 6
- 102000039446 nucleic acids Human genes 0.000 claims abstract 6
- 150000007523 nucleic acids Chemical class 0.000 claims abstract 6
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims abstract 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims abstract 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims abstract 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims abstract 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims abstract 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims abstract 4
- 239000002773 nucleotide Substances 0.000 claims abstract 4
- 125000003729 nucleotide group Chemical group 0.000 claims abstract 4
- 238000003780 insertion Methods 0.000 claims abstract 3
- 230000037431 insertion Effects 0.000 claims abstract 3
- 208000003098 Ganglion Cysts Diseases 0.000 claims abstract 2
- 208000005400 Synovial Cyst Diseases 0.000 claims abstract 2
- 241000700605 Viruses Species 0.000 claims abstract 2
- 210000000411 amacrine cell Anatomy 0.000 claims abstract 2
- 210000003986 cell retinal photoreceptor Anatomy 0.000 claims abstract 2
- 210000002287 horizontal cell Anatomy 0.000 claims abstract 2
- 210000000327 mueller cell Anatomy 0.000 claims abstract 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims abstract 2
- 229920001184 polypeptide Polymers 0.000 claims 11
- 102000004196 processed proteins & peptides Human genes 0.000 claims 11
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 3
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 2
- 108091023037 Aptamer Proteins 0.000 claims 2
- 208000006992 Color Vision Defects Diseases 0.000 claims 2
- 102000007072 Nerve Growth Factors Human genes 0.000 claims 2
- 102000004330 Rhodopsin Human genes 0.000 claims 2
- 108090000820 Rhodopsin Proteins 0.000 claims 2
- 230000001772 anti-angiogenic effect Effects 0.000 claims 2
- 201000007254 color blindness Diseases 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 230000000324 neuroprotective effect Effects 0.000 claims 2
- 239000003900 neurotrophic factor Substances 0.000 claims 2
- 239000000790 retinal pigment Substances 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 229940088872 Apoptosis inhibitor Drugs 0.000 claims 1
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 1
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 1
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 1
- 102400001368 Epidermal growth factor Human genes 0.000 claims 1
- 101800003838 Epidermal growth factor Proteins 0.000 claims 1
- 102000003974 Fibroblast growth factor 2 Human genes 0.000 claims 1
- 108090000379 Fibroblast growth factor 2 Proteins 0.000 claims 1
- 102000013446 GTP Phosphohydrolases Human genes 0.000 claims 1
- 108091006109 GTPases Proteins 0.000 claims 1
- 208000010412 Glaucoma Diseases 0.000 claims 1
- 102000003693 Hedgehog Proteins Human genes 0.000 claims 1
- 108090000031 Hedgehog Proteins Proteins 0.000 claims 1
- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims 1
- 101000668058 Infectious salmon anemia virus (isolate Atlantic salmon/Norway/810/9/99) RNA-directed RNA polymerase catalytic subunit Proteins 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 1
- 208000017442 Retinal disease Diseases 0.000 claims 1
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 1
- 101710111169 Retinoschisin Proteins 0.000 claims 1
- 102100039507 Retinoschisin Human genes 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 201000000761 achromatopsia Diseases 0.000 claims 1
- 230000002424 anti-apoptotic effect Effects 0.000 claims 1
- 239000000158 apoptosis inhibitor Substances 0.000 claims 1
- 210000005013 brain tissue Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 229940116977 epidermal growth factor Drugs 0.000 claims 1
- 208000030533 eye disease Diseases 0.000 claims 1
- 230000002518 glial effect Effects 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 229940053128 nerve growth factor Drugs 0.000 claims 1
- 230000002093 peripheral effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 201000007714 retinoschisis Diseases 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- VBEQCZHXXJYVRD-GACYYNSASA-N uroanthelone Chemical compound C([C@@H](C(=O)N[C@H](C(=O)N[C@@H](CS)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CS)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CS)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)C(C)C)[C@@H](C)O)NC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CC=1NC=NC=1)NC(=O)[C@H](CCSC)NC(=O)[C@H](CS)NC(=O)[C@@H](NC(=O)CNC(=O)CNC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CS)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@H](CO)NC(=O)[C@H]1N(CCC1)C(=O)[C@H](CS)NC(=O)CNC(=O)[C@H]1N(CCC1)C(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC(N)=O)C(C)C)[C@@H](C)CC)C1=CC=C(O)C=C1 VBEQCZHXXJYVRD-GACYYNSASA-N 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
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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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