JP2020511167A5 - - Google Patents
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- Publication number
- JP2020511167A5 JP2020511167A5 JP2020501858A JP2020501858A JP2020511167A5 JP 2020511167 A5 JP2020511167 A5 JP 2020511167A5 JP 2020501858 A JP2020501858 A JP 2020501858A JP 2020501858 A JP2020501858 A JP 2020501858A JP 2020511167 A5 JP2020511167 A5 JP 2020511167A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- vector
- variant
- retinitis pigmentosa
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 150000007523 nucleic acids Chemical group 0.000 claims 30
- 102000039446 nucleic acids Human genes 0.000 claims 22
- 108020004707 nucleic acids Proteins 0.000 claims 22
- 108091033319 polynucleotide Proteins 0.000 claims 21
- 102000040430 polynucleotide Human genes 0.000 claims 21
- 239000002157 polynucleotide Substances 0.000 claims 21
- 239000013598 vector Substances 0.000 claims 14
- 239000013603 viral vector Substances 0.000 claims 14
- 201000011623 retinitis pigmentosa 45 Diseases 0.000 claims 11
- 201000007737 Retinal degeneration Diseases 0.000 claims 9
- 108700019146 Transgenes Proteins 0.000 claims 9
- 230000004258 retinal degeneration Effects 0.000 claims 9
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 8
- 229940123587 Cell cycle inhibitor Drugs 0.000 claims 6
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 239000013612 plasmid Substances 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 5
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 4
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 4
- 241000700584 Simplexvirus Species 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 claims 3
- 241000700605 Viruses Species 0.000 claims 3
- 230000035790 physiological processes and functions Effects 0.000 claims 3
- 230000008488 polyadenylation Effects 0.000 claims 3
- 238000006467 substitution reaction Methods 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 102100024081 Aryl-hydrocarbon-interacting protein-like 1 Human genes 0.000 claims 2
- 102100022440 Battenin Human genes 0.000 claims 2
- 102100022794 Bestrophin-1 Human genes 0.000 claims 2
- 102000014812 CACNA1F Human genes 0.000 claims 2
- 102100035673 Centrosomal protein of 290 kDa Human genes 0.000 claims 2
- 101710198317 Centrosomal protein of 290 kDa Proteins 0.000 claims 2
- 108091006146 Channels Proteins 0.000 claims 2
- 102100031060 Clarin-1 Human genes 0.000 claims 2
- 102100029362 Cone-rod homeobox protein Human genes 0.000 claims 2
- 102100029141 Cyclic nucleotide-gated cation channel beta-1 Human genes 0.000 claims 2
- 102100033270 Cyclin-dependent kinase inhibitor 1 Human genes 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- -1 GP R98 Proteins 0.000 claims 2
- 102100033969 Guanylyl cyclase-activating protein 1 Human genes 0.000 claims 2
- 102100033968 Guanylyl cyclase-activating protein 2 Human genes 0.000 claims 2
- 102100037931 Harmonin Human genes 0.000 claims 2
- 101000594506 Homo sapiens Acyl-coenzyme A diphosphatase NUDT19 Proteins 0.000 claims 2
- 101000833576 Homo sapiens Aryl-hydrocarbon-interacting protein-like 1 Proteins 0.000 claims 2
- 101000901683 Homo sapiens Battenin Proteins 0.000 claims 2
- 101000903449 Homo sapiens Bestrophin-1 Proteins 0.000 claims 2
- 101000992973 Homo sapiens Clarin-1 Proteins 0.000 claims 2
- 101000919370 Homo sapiens Cone-rod homeobox protein Proteins 0.000 claims 2
- 101000771075 Homo sapiens Cyclic nucleotide-gated cation channel beta-1 Proteins 0.000 claims 2
- 101000844721 Homo sapiens Deleted in malignant brain tumors 1 protein Proteins 0.000 claims 2
- 101001068480 Homo sapiens Guanylyl cyclase-activating protein 1 Proteins 0.000 claims 2
- 101001068475 Homo sapiens Guanylyl cyclase-activating protein 2 Proteins 0.000 claims 2
- 101000805947 Homo sapiens Harmonin Proteins 0.000 claims 2
- 101000610652 Homo sapiens Peripherin-2 Proteins 0.000 claims 2
- 101000610551 Homo sapiens Prominin-1 Proteins 0.000 claims 2
- 101000781361 Homo sapiens Protein XRP2 Proteins 0.000 claims 2
- 101000726148 Homo sapiens Protein crumbs homolog 1 Proteins 0.000 claims 2
- 101000726110 Homo sapiens Protein crumbs homolog 2 Proteins 0.000 claims 2
- 101001111714 Homo sapiens RING-box protein 2 Proteins 0.000 claims 2
- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims 2
- 101000609947 Homo sapiens Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit alpha Proteins 0.000 claims 2
- 101000609949 Homo sapiens Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit beta Proteins 0.000 claims 2
- 101001106432 Homo sapiens Rod outer segment membrane protein 1 Proteins 0.000 claims 2
- 101000901226 Homo sapiens S-arrestin Proteins 0.000 claims 2
- 101000805943 Homo sapiens Usher syndrome type-1G protein Proteins 0.000 claims 2
- 101000867848 Homo sapiens Voltage-dependent L-type calcium channel subunit alpha-1F Proteins 0.000 claims 2
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 claims 2
- 101000883219 Homo sapiens cGMP-gated cation channel alpha-1 Proteins 0.000 claims 2
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 2
- 108010009047 Myosin VIIa Proteins 0.000 claims 2
- 102000026889 Myosin VIIa Human genes 0.000 claims 2
- 102100034268 Neural retina-specific leucine zipper protein Human genes 0.000 claims 2
- 101710181914 Neural retina-specific leucine zipper protein Proteins 0.000 claims 2
- 102100040375 Peripherin-2 Human genes 0.000 claims 2
- 102100040120 Prominin-1 Human genes 0.000 claims 2
- 102100033154 Protein XRP2 Human genes 0.000 claims 2
- 102100027331 Protein crumbs homolog 1 Human genes 0.000 claims 2
- 102100027317 Protein crumbs homolog 2 Human genes 0.000 claims 2
- 101001000628 Rattus norvegicus Peripheral myelin protein 22 Proteins 0.000 claims 2
- 108050002653 Retinoblastoma protein Proteins 0.000 claims 2
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 2
- 102100040756 Rhodopsin Human genes 0.000 claims 2
- 102100039177 Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit alpha Human genes 0.000 claims 2
- 102100039174 Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit beta Human genes 0.000 claims 2
- 102100021424 Rod outer segment membrane protein 1 Human genes 0.000 claims 2
- 102100022135 S-arrestin Human genes 0.000 claims 2
- 101000733871 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) 60S ribosomal protein L4-A Proteins 0.000 claims 2
- 101000733875 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) 60S ribosomal protein L4-B Proteins 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 101000942604 Sphingomonas wittichii (strain DC-6 / KACC 16600) Chloroacetanilide N-alkylformylase, oxygenase component Proteins 0.000 claims 2
- 102000006601 Thymidine Kinase Human genes 0.000 claims 2
- 108020004440 Thymidine kinase Proteins 0.000 claims 2
- 102100037929 Usher syndrome type-1G protein Human genes 0.000 claims 2
- 206010046865 Vaccinia virus infection Diseases 0.000 claims 2
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 claims 2
- 102100038623 cGMP-gated cation channel alpha-1 Human genes 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 239000004568 cement Substances 0.000 claims 2
- 125000004122 cyclic group Chemical group 0.000 claims 2
- 230000001419 dependent effect Effects 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 208000015181 infectious disease Diseases 0.000 claims 2
- 230000002458 infectious effect Effects 0.000 claims 2
- 108091070501 miRNA Proteins 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 claims 2
- 230000001177 retroviral effect Effects 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- 239000003053 toxin Substances 0.000 claims 2
- 231100000765 toxin Toxicity 0.000 claims 2
- 108700012359 toxins Proteins 0.000 claims 2
- 241000701161 unidentified adenovirus Species 0.000 claims 2
- 241001529453 unidentified herpesvirus Species 0.000 claims 2
- 208000007089 vaccinia Diseases 0.000 claims 2
- 201000004569 Blindness Diseases 0.000 claims 1
- 102100032220 Calcium and integrin-binding family member 2 Human genes 0.000 claims 1
- 108010036281 Cyclic Nucleotide-Gated Cation Channels Proteins 0.000 claims 1
- 102000012003 Cyclic Nucleotide-Gated Cation Channels Human genes 0.000 claims 1
- 102100033233 Cyclin-dependent kinase inhibitor 1B Human genes 0.000 claims 1
- 206010064571 Gene mutation Diseases 0.000 claims 1
- 101000943456 Homo sapiens Calcium and integrin-binding family member 2 Proteins 0.000 claims 1
- 101000944380 Homo sapiens Cyclin-dependent kinase inhibitor 1 Proteins 0.000 claims 1
- 101000944361 Homo sapiens Cyclin-dependent kinase inhibitor 1B Proteins 0.000 claims 1
- 101000944365 Homo sapiens Cyclin-dependent kinase inhibitor 1C Proteins 0.000 claims 1
- 208000032578 Inherited retinal disease Diseases 0.000 claims 1
- 208000001140 Night Blindness Diseases 0.000 claims 1
- 208000017442 Retinal disease Diseases 0.000 claims 1
- 208000032430 Retinal dystrophy Diseases 0.000 claims 1
- 108091006627 SLC12A9 Proteins 0.000 claims 1
- 108700026226 TATA Box Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 230000003796 beauty Effects 0.000 claims 1
- 230000004663 cell proliferation Effects 0.000 claims 1
- 230000007547 defect Effects 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 201000006321 fundus dystrophy Diseases 0.000 claims 1
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims 1
- 239000003999 initiator Substances 0.000 claims 1
- 239000000651 prodrug Substances 0.000 claims 1
- 229940002612 prodrug Drugs 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762474409P | 2017-03-21 | 2017-03-21 | |
| US62/474,409 | 2017-03-21 | ||
| PCT/IB2018/051905 WO2018172961A1 (en) | 2017-03-21 | 2018-03-21 | Gene therapy for the treatment of cngb1-linked retinitis pigmentosa |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020511167A JP2020511167A (ja) | 2020-04-16 |
| JP2020511167A5 true JP2020511167A5 (enExample) | 2021-04-22 |
| JP7257377B2 JP7257377B2 (ja) | 2023-04-13 |
Family
ID=62044771
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020501858A Active JP7257377B2 (ja) | 2017-03-21 | 2018-03-21 | Ｃｎｇｂ１連鎖性網膜色素変性症の治療のための遺伝子療法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12246072B2 (enExample) |
| EP (1) | EP3600446A1 (enExample) |
| JP (1) | JP7257377B2 (enExample) |
| CN (1) | CN111050805A (enExample) |
| AR (1) | AR111188A1 (enExample) |
| AU (1) | AU2018238422B2 (enExample) |
| CA (1) | CA3056211A1 (enExample) |
| TW (1) | TW201902911A (enExample) |
| UY (1) | UY37636A (enExample) |
| WO (1) | WO2018172961A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220175969A1 (en) * | 2019-03-04 | 2022-06-09 | University Of Florida Research Foundation, Incorporated | Enhanced human opsin promoter for rod specific expression |
| WO2020182722A1 (en) * | 2019-03-08 | 2020-09-17 | Universite Paris-Saclay | Improved therapeutic method for rare ocular diseases by gene replacement |
| CN110241202A (zh) * | 2019-06-25 | 2019-09-17 | 复旦大学附属眼耳鼻喉科医院 | 视网膜色素变性突变位点及其应用 |
| JP2022549138A (ja) | 2019-09-18 | 2022-11-24 | インターガラクティック セラピューティクス インコーポレイテッド | 合成ｄｎａベクターおよびその使用法 |
| WO2021146625A1 (en) * | 2020-01-17 | 2021-07-22 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Gene therapy for treatment of crx-autosomal dominant retinopathies |
| US20230174598A1 (en) * | 2020-03-18 | 2023-06-08 | University Of Florida Research Foundation, Incorporated | Modified arrestin-1 to enhance photoreceptor survival in retinal disease |
| CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
| JP2023548746A (ja) * | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| CN113201556B (zh) * | 2021-05-14 | 2022-11-22 | 浙江大学 | pSOY19-ZM2载体、其制备方法及应用 |
| WO2023280388A1 (en) * | 2021-07-06 | 2023-01-12 | Institute Of Molecular And Clinical Ophthalmology Basel | Promoter for specific expression of genes in rod photoreceptors |
| CN115820653A (zh) * | 2021-09-17 | 2023-03-21 | 深圳华大生命科学研究院 | 基因突变体及其应用 |
| CN119432856B (zh) * | 2024-12-30 | 2025-08-12 | 云舟生物科技(广州)股份有限公司 | 核酸分子、病毒颗粒及其制备方法和应用 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2140004B1 (en) * | 2007-04-12 | 2022-01-12 | The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin | Genetic suppression and replacement |
| US11246947B2 (en) | 2013-08-05 | 2022-02-15 | Academisch Ziekenhuis Leiden H.O.D. N. Leids Universitair Medisch Centrum | Recombinant AAV-crumbs homologue composition and methods for treating LCA-8 and progressive RP |
| ES2760263T3 (es) * | 2014-03-21 | 2020-05-13 | Genzyme Corp | Tratamiento génico de la retinitis pigmentaria |
| EP3210632B1 (en) * | 2016-02-23 | 2022-09-28 | EyeServ GmbH | Gene therapy for the treatment of a retinal degeneration disease |
-
2018
- 2018-03-21 WO PCT/IB2018/051905 patent/WO2018172961A1/en not_active Ceased
- 2018-03-21 AU AU2018238422A patent/AU2018238422B2/en active Active
- 2018-03-21 CN CN201880031841.5A patent/CN111050805A/zh active Pending
- 2018-03-21 UY UY0001037636A patent/UY37636A/es not_active Application Discontinuation
- 2018-03-21 CA CA3056211A patent/CA3056211A1/en active Pending
- 2018-03-21 EP EP18719652.2A patent/EP3600446A1/en active Pending
- 2018-03-21 TW TW107109700A patent/TW201902911A/zh unknown
- 2018-03-21 AR ARP180100661A patent/AR111188A1/es unknown
- 2018-03-21 US US16/495,826 patent/US12246072B2/en active Active
- 2018-03-21 JP JP2020501858A patent/JP7257377B2/ja active Active
-
2025
- 2025-02-03 US US19/043,744 patent/US20250262328A1/en active Pending
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