JP2019529385A5 - - Google Patents
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- Publication number
- JP2019529385A5 JP2019529385A5 JP2019512292A JP2019512292A JP2019529385A5 JP 2019529385 A5 JP2019529385 A5 JP 2019529385A5 JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019529385 A5 JP2019529385 A5 JP 2019529385A5
- Authority
- JP
- Japan
- Prior art keywords
- use according
- mammal
- cells
- cns
- therapeutic protein
- 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
- 210000003169 central nervous system Anatomy 0.000 claims 16
- 230000001225 therapeutic effect Effects 0.000 claims 16
- 210000004027 cell Anatomy 0.000 claims 15
- 102000004169 proteins and genes Human genes 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 15
- 210000001519 tissue Anatomy 0.000 claims 15
- 241000124008 Mammalia Species 0.000 claims 14
- 210000000056 organ Anatomy 0.000 claims 12
- 108010025628 Apolipoproteins E Proteins 0.000 claims 7
- 102000013918 Apolipoproteins E Human genes 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 7
- 102000001708 Protein Isoforms Human genes 0.000 claims 7
- 108010029485 Protein Isoforms Proteins 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 239000003814 drug Substances 0.000 claims 6
- 239000002245 particle Substances 0.000 claims 6
- 229940124597 therapeutic agent Drugs 0.000 claims 6
- 210000005252 bulbus oculi Anatomy 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 230000001681 protective effect Effects 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims 4
- 101000901683 Homo sapiens Battenin Proteins 0.000 claims 3
- 101000710215 Homo sapiens Ceroid-lipofuscinosis neuronal protein 6 Proteins 0.000 claims 3
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 3
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims 3
- CTMZLDSMFCVUNX-VMIOUTBZSA-N cytidylyl-(3'->5')-guanosine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](OP(O)(=O)OC[C@@H]2[C@H]([C@@H](O)[C@@H](O2)N2C3=C(C(N=C(N)N3)=O)N=C2)O)[C@@H](CO)O1 CTMZLDSMFCVUNX-VMIOUTBZSA-N 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 3
- 210000003463 organelle Anatomy 0.000 claims 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
- 102000053187 Glucuronidase Human genes 0.000 claims 2
- 108010060309 Glucuronidase Proteins 0.000 claims 2
- 101000710213 Homo sapiens Protein CLN8 Proteins 0.000 claims 2
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 claims 2
- 102000005327 Palmitoyl protein thioesterase Human genes 0.000 claims 2
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims 2
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims 2
- 230000009286 beneficial effect Effects 0.000 claims 2
- 210000001508 eye Anatomy 0.000 claims 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 108010060219 Apolipoprotein E2 Proteins 0.000 claims 1
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims 1
- 101000574223 Homo sapiens Palmitoyl-protein thioesterase 1 Proteins 0.000 claims 1
- 101100261165 Homo sapiens TPP1 gene Proteins 0.000 claims 1
- 101100426960 Homo sapiens TTPA gene Proteins 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 241001465754 Metazoa Species 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 1
- 208000017478 adult neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 230000003542 behavioural effect Effects 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 230000008021 deposition Effects 0.000 claims 1
- 210000003890 endocrine cell Anatomy 0.000 claims 1
- 239000000945 filler Substances 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 102000056242 human CLN3 Human genes 0.000 claims 1
- 102000045627 human CLN6 Human genes 0.000 claims 1
- 102000051672 human PPT1 Human genes 0.000 claims 1
- 210000005228 liver tissue Anatomy 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000004923 pancreatic tissue Anatomy 0.000 claims 1
- 230000007310 pathophysiology Effects 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000008092 positive effect Effects 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022002163A JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662383274P | 2016-09-02 | 2016-09-02 | |
| US62/383,274 | 2016-09-02 | ||
| PCT/US2017/049959 WO2018045347A1 (en) | 2016-09-02 | 2017-09-01 | Methods and vectors for treating cns disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002163A Division JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019529385A JP2019529385A (ja) | 2019-10-17 |
| JP2019529385A5 true JP2019529385A5 (enExample) | 2020-09-17 |
| JP7610923B2 JP7610923B2 (ja) | 2025-01-09 |
Family
ID=61309371
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019512292A Active JP7610923B2 (ja) | 2016-09-02 | 2017-09-01 | Ｃｎｓ障害を治療するための方法及びベクター |
| JP2022002163A Pending JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002163A Pending JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190192693A1 (enExample) |
| EP (1) | EP3506930A4 (enExample) |
| JP (2) | JP7610923B2 (enExample) |
| CN (1) | CN110121356A (enExample) |
| AU (2) | AU2017318717B2 (enExample) |
| BR (1) | BR112019004353A2 (enExample) |
| CA (1) | CA3035628A1 (enExample) |
| MX (1) | MX2019002518A (enExample) |
| WO (1) | WO2018045347A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| MX2020000713A (es) * | 2017-07-17 | 2020-08-31 | Spark Therapeutics Inc | Metodos de aferesis y sus usos. |
| US12275948B2 (en) | 2018-06-28 | 2025-04-15 | The University Of North Carolina At Chapel Hill | Optimized CLN5 genes and expression cassettes and their use |
| JP7624724B2 (ja) | 2018-09-26 | 2025-01-31 | カリフォルニア インスティテュート オブ テクノロジー | 標的遺伝子療法のためのアデノ随伴ウイルス組成物 |
| JP2022513034A (ja) * | 2018-11-14 | 2022-02-07 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| JP7637058B2 (ja) | 2019-02-22 | 2025-02-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| US20220348638A1 (en) * | 2019-09-24 | 2022-11-03 | The J. David Gladstone Institutes, a testamentary trust estabilished under the Will of J. David Glad | Method of targeting neuronal apoe to treat a neurocognitive disorder |
| MX2022004524A (es) * | 2019-10-16 | 2022-07-21 | Univ Cornell | Tratamiento génico para la enfermedad de alzheimer. |
| AU2021351720A1 (en) * | 2020-10-01 | 2023-05-11 | Sangamo Therapeutics, Inc. | Methods of detecting anti-aav antibodies |
| CN112852881B (zh) * | 2021-02-09 | 2023-08-15 | 南京贝思奥生物科技有限公司 | 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法 |
| TW202426479A (zh) * | 2022-10-10 | 2024-07-01 | 美商星火治療公司 | Apoe基因療法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7135339B2 (en) * | 2003-11-20 | 2006-11-14 | University Of Iowa Research Foundation | Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV) |
| JP5704361B2 (ja) * | 2010-10-27 | 2015-04-22 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| IN2014KN02672A (enExample) * | 2012-05-18 | 2015-05-08 | Univ Iowa Res Found | |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| CA2931220A1 (en) * | 2013-11-20 | 2015-05-28 | University Of Iowa Research Foundation | Methods and compositions for treating amyloid deposits |
| AU2016219789B2 (en) * | 2015-02-20 | 2021-03-25 | Fondazione Telethon | Methods and compositions for treating genetic eye diseases |
-
2017
- 2017-09-01 CN CN201780067677.9A patent/CN110121356A/zh active Pending
- 2017-09-01 WO PCT/US2017/049959 patent/WO2018045347A1/en not_active Ceased
- 2017-09-01 JP JP2019512292A patent/JP7610923B2/ja active Active
- 2017-09-01 AU AU2017318717A patent/AU2017318717B2/en active Active
- 2017-09-01 EP EP17847658.6A patent/EP3506930A4/en active Pending
- 2017-09-01 MX MX2019002518A patent/MX2019002518A/es unknown
- 2017-09-01 US US16/329,697 patent/US20190192693A1/en not_active Abandoned
- 2017-09-01 CA CA3035628A patent/CA3035628A1/en active Pending
- 2017-09-01 BR BR112019004353A patent/BR112019004353A2/pt unknown
-
2022
- 2022-01-11 JP JP2022002163A patent/JP2022050574A/ja active Pending
-
2024
- 2024-10-06 AU AU2024227138A patent/AU2024227138A1/en not_active Abandoned
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