RU2018142273A - Способ улучшения опосредованной вирусами доставки генов в глаз с использованием ингибиторов протеасом - Google Patents
Способ улучшения опосредованной вирусами доставки генов в глаз с использованием ингибиторов протеасом Download PDFInfo
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- RU2018142273A RU2018142273A RU2018142273A RU2018142273A RU2018142273A RU 2018142273 A RU2018142273 A RU 2018142273A RU 2018142273 A RU2018142273 A RU 2018142273A RU 2018142273 A RU2018142273 A RU 2018142273A RU 2018142273 A RU2018142273 A RU 2018142273A
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- eye
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- viral vector
- proteasome inhibitor
- opsin
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- 238000000034 method Methods 0.000 title claims 20
- 108090000623 proteins and genes Proteins 0.000 title claims 5
- 239000003112 inhibitor Substances 0.000 title 1
- 239000013603 viral vector Substances 0.000 claims 8
- 229940079156 Proteasome inhibitor Drugs 0.000 claims 7
- 239000003207 proteasome inhibitor Substances 0.000 claims 7
- 102000010175 Opsin Human genes 0.000 claims 6
- 108050001704 Opsin Proteins 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- AOJJSUZBOXZQNB-TZSSRYMLSA-N Doxorubicin Chemical compound O([C@H]1C[C@@](O)(CC=2C(O)=C3C(=O)C=4C=CC=C(C=4C(=O)C3=C(O)C=21)OC)C(=O)CO)[C@H]1C[C@H](N)[C@H](O)[C@H](C)O1 AOJJSUZBOXZQNB-TZSSRYMLSA-N 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 230000002207 retinal effect Effects 0.000 claims 4
- -1 epoxyimycin Chemical compound 0.000 claims 3
- 108010082845 Bacteriorhodopsins Proteins 0.000 claims 2
- 108010035848 Channelrhodopsins Proteins 0.000 claims 2
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 2
- 206010061218 Inflammation Diseases 0.000 claims 2
- 102100025912 Melanopsin Human genes 0.000 claims 2
- 201000000582 Retinoblastoma Diseases 0.000 claims 2
- 206010038926 Retinopathy hypertensive Diseases 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- AUZONCFQVSMFAP-UHFFFAOYSA-N disulfiram Chemical compound CCN(CC)C(=S)SSC(=S)N(CC)CC AUZONCFQVSMFAP-UHFFFAOYSA-N 0.000 claims 2
- 229960004679 doxorubicin Drugs 0.000 claims 2
- 201000001948 hypertensive retinopathy Diseases 0.000 claims 2
- 230000004054 inflammatory process Effects 0.000 claims 2
- 108010005417 melanopsin Proteins 0.000 claims 2
- 201000002575 ocular melanoma Diseases 0.000 claims 2
- 210000000056 organ Anatomy 0.000 claims 2
- 210000001519 tissue Anatomy 0.000 claims 2
- WMBWREPUVVBILR-WIYYLYMNSA-N (-)-Epigallocatechin-3-o-gallate Chemical compound O([C@@H]1CC2=C(O)C=C(C=C2O[C@@H]1C=1C=C(O)C(O)=C(O)C=1)O)C(=O)C1=CC(O)=C(O)C(O)=C1 WMBWREPUVVBILR-WIYYLYMNSA-N 0.000 claims 1
- 229930014124 (-)-epigallocatechin gallate Natural products 0.000 claims 1
- AXFYFNCPONWUHW-UHFFFAOYSA-N 3-hydroxyisovaleric acid Chemical compound CC(C)(O)CC(O)=O AXFYFNCPONWUHW-UHFFFAOYSA-N 0.000 claims 1
- WMBWREPUVVBILR-UHFFFAOYSA-N GCG Natural products C=1C(O)=C(O)C(O)=CC=1C1OC2=CC(O)=CC(O)=C2CC1OC(=O)C1=CC(O)=C(O)C(O)=C1 WMBWREPUVVBILR-UHFFFAOYSA-N 0.000 claims 1
- DAQAKHDKYAWHCG-UHFFFAOYSA-N Lactacystin Natural products CC(=O)NC(C(O)=O)CSC(=O)C1(C(O)C(C)C)NC(=O)C(C)C1O DAQAKHDKYAWHCG-UHFFFAOYSA-N 0.000 claims 1
- TZYWCYJVHRLUCT-VABKMULXSA-N N-benzyloxycarbonyl-L-leucyl-L-leucyl-L-leucinal Chemical compound CC(C)C[C@@H](C=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(C)C)NC(=O)OCC1=CC=CC=C1 TZYWCYJVHRLUCT-VABKMULXSA-N 0.000 claims 1
- 206010034972 Photosensitivity reaction Diseases 0.000 claims 1
- USZYSDMBJDPRIF-SVEJIMAYSA-N aclacinomycin A Chemical compound O([C@H]1[C@@H](O)C[C@@H](O[C@H]1C)O[C@H]1[C@H](C[C@@H](O[C@H]1C)O[C@H]1C[C@]([C@@H](C2=CC=3C(=O)C4=CC=CC(O)=C4C(=O)C=3C(O)=C21)C(=O)OC)(O)CC)N(C)C)[C@H]1CCC(=O)[C@H](C)O1 USZYSDMBJDPRIF-SVEJIMAYSA-N 0.000 claims 1
- 229960004176 aclarubicin Drugs 0.000 claims 1
- 210000000411 amacrine cell Anatomy 0.000 claims 1
- GXJABQQUPOEUTA-RDJZCZTQSA-N bortezomib Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)B(O)O)NC(=O)C=1N=CC=NC=1)C1=CC=CC=C1 GXJABQQUPOEUTA-RDJZCZTQSA-N 0.000 claims 1
- 229960001467 bortezomib Drugs 0.000 claims 1
- BLMPQMFVWMYDKT-NZTKNTHTSA-N carfilzomib Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)N[C@@H](CC(C)C)C(=O)[C@]1(C)OC1)NC(=O)CN1CCOCC1)CC1=CC=CC=C1 BLMPQMFVWMYDKT-NZTKNTHTSA-N 0.000 claims 1
- 229960002438 carfilzomib Drugs 0.000 claims 1
- 108010021331 carfilzomib Proteins 0.000 claims 1
- SJFBTAPEPRWNKH-CCKFTAQKSA-N delanzomib Chemical compound CC(C)C[C@@H](B(O)O)NC(=O)[C@H]([C@@H](C)O)NC(=O)C1=CC=CC(C=2C=CC=CC=2)=N1 SJFBTAPEPRWNKH-CCKFTAQKSA-N 0.000 claims 1
- 229950001466 delanzomib Drugs 0.000 claims 1
- 229960002563 disulfiram Drugs 0.000 claims 1
- 230000001434 glomerular Effects 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- DAQAKHDKYAWHCG-RWTHQLGUSA-N lactacystin Chemical compound CC(=O)N[C@H](C(O)=O)CSC(=O)[C@]1([C@@H](O)C(C)C)NC(=O)[C@H](C)[C@@H]1O DAQAKHDKYAWHCG-RWTHQLGUSA-N 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 229950002736 marizomib Drugs 0.000 claims 1
- 210000000327 mueller cell Anatomy 0.000 claims 1
- SWZXEVABPLUDIO-WSZYKNRRSA-N n-[(2s)-3-methoxy-1-[[(2s)-3-methoxy-1-[[(2s)-1-[(2r)-2-methyloxiran-2-yl]-1-oxo-3-phenylpropan-2-yl]amino]-1-oxopropan-2-yl]amino]-1-oxopropan-2-yl]-2-methyl-1,3-thiazole-5-carboxamide Chemical compound N([C@@H](COC)C(=O)N[C@@H](COC)C(=O)N[C@@H](CC=1C=CC=CC=1)C(=O)[C@]1(C)OC1)C(=O)C1=CN=C(C)S1 SWZXEVABPLUDIO-WSZYKNRRSA-N 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 210000000608 photoreceptor cell Anatomy 0.000 claims 1
- 230000036211 photosensitivity Effects 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 210000003994 retinal ganglion cell Anatomy 0.000 claims 1
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims 1
- NGWSFRIPKNWYAO-UHFFFAOYSA-N salinosporamide A Natural products N1C(=O)C(CCCl)C2(C)OC(=O)C21C(O)C1CCCC=C1 NGWSFRIPKNWYAO-UHFFFAOYSA-N 0.000 claims 1
- NGWSFRIPKNWYAO-SHTIJGAHSA-N salinosporamide A Chemical compound C([C@@H]1[C@H](O)[C@]23C(=O)O[C@]2([C@H](C(=O)N3)CCCl)C)CCC=C1 NGWSFRIPKNWYAO-SHTIJGAHSA-N 0.000 claims 1
- 210000004127 vitreous body Anatomy 0.000 claims 1
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Claims (18)
1. Способ улучшения доставки представляющего интерес гена в глаз субъекта, включающий введение ингибитора протеасомы и вирусного вектора, кодирующего представляющий интерес ген, в глаз.
2. Способ по п. 1, где ингибитор протеасомы представляет собой доксорубицин (DOX), акларубицин, бортезомиб, лактацистин, дисульфирам, эпигаллокатехин-3-галлат, маризомиб (салиноспорамид A), опрозомиб (ONX-0912), деланзомиб (CEP-18770), эпоксимицин, MG132, бета-гидрокси-бета-метилбутират или карфилзомиб.
3. Способ по п. 1, где представляющий интерес ген представляет собой опсин.
4. Способ по п. 3, где опсин выбран из группы, состоящей из канального родопсина, галородопсина, меланопсина, опсина пинеального органа, бактериородопсина и протеородопсина, или их функционального варианта.
5. Способ по п. 1, где представляющий интерес ген является функционально связанным со специфическим для клеток промотором.
6. Способ по п. 1, где вирусный вектор является инкапсулированным в наночастицу, полимер или липосому.
7. Способ по п. 1, где субъект страдает от офтальмологического заболевания или нарушения.
8. Способ по п. 7, где офтальмологическое заболевание представляет собой ретинобластому, меланому глаза, диабетическую ретинопатию, гипертензивную ретинопатию или воспаление тканей глаза.
9. Способ по п. 1, где ингибитор протеасомы и вирусный вектор доставляют одновременно или последовательно.
10. Способ по п. 1, где вирусный вектор доставляют в клетку сетчатки.
11. Способ по п. 10, где клетка сетчатки представляет собой ганглионарную клетку сетчатки, биполярную клетку сетчатки, горизонтальную клетку сетчатки, амакриновую клетку, фоторецепторную клетку, глиальную клетку Мюллера или клетку пигментного эпителия сетчатки.
12. Способ по п. 1 или 9, где ингибитор протеасомы и вирусный вектор вводят в стекловидное тело глаза.
13. Способ по п. 1 или 9, где ингибитор протеасомы и вирусный вектор вводят способом, при котором введение проводят посредством инъекции или инфузии.
14. Способ по п. 1 или 9, где ингибитор протеасомы и вирусный вектор вводят не субретинально.
15. Способ увеличения светочувствительности или улучшения или восстановления зрения у субъекта, включающий введение ингибитора протеасомы и вирусного вектора, который кодирует опсин, в стекловидное тело глаза.
16. Способ по п. 15, где указанный опсин выбран из группы, состоящей из канального родопсина, галородопсина, меланопсина, опсина пинеального органа, бактериородопсина и протеородопсина, или их функционального варианта.
17. Способ по п. 15, где субъект имеет офтальмологическое заболевание или нарушение.
18. Способ по любому из пп. 15-17, где офтальмологическое заболевание представляет собой ретинобластому, меланому глаза, диабетическую ретинопатию, гипертензивную ретинопатию или воспаление тканей глаза.
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| PCT/US2017/030908 WO2017192764A1 (en) | 2016-05-03 | 2017-05-03 | Method of enhancing viral-mediated gene delivery in the eye using proteosome inhibitors |
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| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| FR2777909B1 (fr) | 1998-04-24 | 2002-08-02 | Pasteur Institut | Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex |
| CA2346262A1 (en) | 1998-09-17 | 2000-03-23 | University Of Florida | Methods for treatment of degenerative retinal diseases |
| DE60234824D1 (de) | 2001-05-01 | 2010-02-04 | Ca Nat Research Council | Induzierbares expressionssystem in eukaryotischen zellen |
| WO2002101012A2 (en) * | 2001-06-08 | 2002-12-19 | Children's Hospital Research Foundation | Regulation of transgene expression following aav transduction |
| JP2006521825A (ja) * | 2003-03-31 | 2006-09-28 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | ｒＡＡＶ導入を高めるための化合物および方法 |
| AU2007247929A1 (en) * | 2006-05-04 | 2007-11-15 | Pennsylvania College Of Optometry | Restoration of visual responses by In Vivo delivery of rhodopsin nucleic acids |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| MX2010012592A (es) * | 2008-05-20 | 2011-05-05 | Eos Neuroscience Inc | Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas. |
| JP6141021B2 (ja) * | 2010-02-05 | 2017-06-07 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | パルボウイルスの形質導入を増強するための組成物および方法 |
| HUE040487T2 (hu) | 2012-03-05 | 2019-03-28 | Univ Wayne State | Csatornarodopszin-2 (CHOP2) mutációk azonosítása és alkalmazási eljárások |
| TWI698240B (zh) * | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| CA2942324C (en) | 2014-03-11 | 2024-05-14 | Wayne State University | A modified mglur6 promoter and methods of use |
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| US20170319669A1 (en) | 2017-11-09 |
| KR20230160967A (ko) | 2023-11-24 |
| IL262679A (en) | 2018-12-31 |
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