JP2019535645A5 - - Google Patents
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- Publication number
- JP2019535645A5 JP2019535645A5 JP2019513898A JP2019513898A JP2019535645A5 JP 2019535645 A5 JP2019535645 A5 JP 2019535645A5 JP 2019513898 A JP2019513898 A JP 2019513898A JP 2019513898 A JP2019513898 A JP 2019513898A JP 2019535645 A5 JP2019535645 A5 JP 2019535645A5
- Authority
- JP
- Japan
- Prior art keywords
- chimeric polypeptide
- cell
- polypeptide according
- seq
- amino acid
- 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.)
- Pending
Links
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 188
- 229920001184 polypeptide Polymers 0.000 claims description 187
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 187
- 210000004027 cell Anatomy 0.000 claims description 44
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 32
- 102000004139 alpha-Amylases Human genes 0.000 claims description 30
- 108090000637 alpha-Amylases Proteins 0.000 claims description 30
- 229940024171 alpha-amylase Drugs 0.000 claims description 30
- 238000000034 method Methods 0.000 claims description 18
- 239000000427 antigen Substances 0.000 claims description 16
- 108091007433 antigens Proteins 0.000 claims description 16
- 102000036639 antigens Human genes 0.000 claims description 16
- 239000012634 fragment Substances 0.000 claims description 16
- 102000039446 nucleic acids Human genes 0.000 claims description 16
- 108020004707 nucleic acids Proteins 0.000 claims description 16
- 150000007523 nucleic acids Chemical class 0.000 claims description 16
- 210000000663 muscle cell Anatomy 0.000 claims description 11
- 210000004962 mammalian cell Anatomy 0.000 claims description 10
- 210000002569 neuron Anatomy 0.000 claims description 10
- 239000002773 nucleotide Substances 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 208000005870 Lafora disease Diseases 0.000 claims description 7
- 208000014161 Lafora myoclonic epilepsy Diseases 0.000 claims description 7
- 108020001507 fusion proteins Proteins 0.000 claims description 6
- 102000037865 fusion proteins Human genes 0.000 claims description 6
- 230000003301 hydrolyzing effect Effects 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- 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 description 4
- 150000001413 amino acids Chemical class 0.000 claims description 4
- 210000004958 brain cell Anatomy 0.000 claims description 4
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 4
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 4
- 238000000338 in vitro Methods 0.000 claims description 4
- 210000000107 myocyte Anatomy 0.000 claims description 3
- 241000271566 Aves Species 0.000 claims description 2
- 125000001433 C-terminal amino-acid group Chemical group 0.000 claims description 2
- 108010078791 Carrier Proteins Proteins 0.000 claims description 2
- 102100021468 Equilibrative nucleoside transporter 2 Human genes 0.000 claims description 2
- 241000238631 Hexapoda Species 0.000 claims description 2
- 101000822017 Homo sapiens Equilibrative nucleoside transporter 2 Proteins 0.000 claims description 2
- 125000001429 N-terminal alpha-amino-acid group Chemical group 0.000 claims description 2
- 102000019055 Nucleoside Transport Proteins Human genes 0.000 claims description 2
- 210000004556 brain Anatomy 0.000 claims description 2
- 210000004671 cell-free system Anatomy 0.000 claims description 2
- 230000000694 effects Effects 0.000 claims description 2
- 230000013595 glycosylation Effects 0.000 claims description 2
- 238000006206 glycosylation reaction Methods 0.000 claims description 2
- 108091006527 nucleoside transporters Proteins 0.000 claims description 2
- 210000001236 prokaryotic cell Anatomy 0.000 claims description 2
- 210000005253 yeast cell Anatomy 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 15
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662385656P | 2016-09-09 | 2016-09-09 | |
| US62/385,656 | 2016-09-09 | ||
| US201762553048P | 2017-08-31 | 2017-08-31 | |
| US62/553,048 | 2017-08-31 | ||
| PCT/US2017/050787 WO2018049237A1 (en) | 2016-09-09 | 2017-09-08 | Methods and compositions for treatment of lafora disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019535645A JP2019535645A (ja) | 2019-12-12 |
| JP2019535645A5 true JP2019535645A5 (enExample) | 2020-10-22 |
Family
ID=61562328
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019513898A Pending JP2019535645A (ja) | 2016-09-09 | 2017-09-08 | ラフォラ病の処置のための方法及び組成物 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10781433B2 (enExample) |
| EP (1) | EP3509614A4 (enExample) |
| JP (1) | JP2019535645A (enExample) |
| CN (1) | CN109963576A (enExample) |
| AU (1) | AU2017322483A1 (enExample) |
| CA (1) | CA3036524A1 (enExample) |
| WO (1) | WO2018049237A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9701740B2 (en) | 2011-04-01 | 2017-07-11 | Yale University | Cell-penetrating anti-DNA antibodies and uses thereof inhibit DNA repair |
| WO2017218824A1 (en) | 2016-06-15 | 2017-12-21 | Yale University | Anti-guanosine antibody as a molecular delivery vehicle |
| EP3471777A1 (en) | 2016-06-15 | 2019-04-24 | Yale University | Antibody-mediated autocatalytic, targeted delivery of nanocarriers to tumors |
| KR20220049568A (ko) * | 2019-08-22 | 2022-04-21 | 더 리전츠 오브 더 유니버시티 오브 캘리포니아 | 엔젤만 증후군의 치료를 위한 ube3a |
| KR102814185B1 (ko) | 2023-07-20 | 2025-06-12 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| WO2025018822A1 (ko) | 2023-07-20 | 2025-01-23 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6166041B2 (ja) | 2009-06-15 | 2017-07-19 | バレリオン セラピューティクス, エルエルシー | ミオチューブラリン1(mtm1)ポリペプチドを含むキメラポリペプチドを使用して筋細管ミオパシーを処置するための方法および組成物 |
| US20130273026A1 (en) | 2012-04-12 | 2013-10-17 | Integrative Enzymatics, Inc. | Composition and method for modulating inflammatory molecules with amylase |
| HK1220980A1 (zh) * | 2013-02-20 | 2017-05-19 | Valerion Therapeutics, Llc | 用於治疗庞贝氏症的方法和组合物 |
| JP2016513114A (ja) * | 2013-02-20 | 2016-05-12 | バレリオン セラピューティクス, エルエルシー | フォーズス・コリ病の処置のための方法および組成物 |
| US10221250B2 (en) * | 2014-01-13 | 2019-03-05 | Valerion Therapeutics, Llc | Internalizing moieties |
| WO2015192092A1 (en) * | 2014-06-13 | 2015-12-17 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
| US10501554B2 (en) * | 2014-08-27 | 2019-12-10 | Valerion Therapeutics, Llc | Internalizing moieties for treatment of cancer |
-
2017
- 2017-09-08 WO PCT/US2017/050787 patent/WO2018049237A1/en not_active Ceased
- 2017-09-08 US US16/332,331 patent/US10781433B2/en active Active
- 2017-09-08 CA CA3036524A patent/CA3036524A1/en not_active Abandoned
- 2017-09-08 AU AU2017322483A patent/AU2017322483A1/en not_active Abandoned
- 2017-09-08 CN CN201780069052.6A patent/CN109963576A/zh active Pending
- 2017-09-08 EP EP17849664.2A patent/EP3509614A4/en not_active Withdrawn
- 2017-09-08 JP JP2019513898A patent/JP2019535645A/ja active Pending
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