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 (https=) | 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 (https=) |
| EP (1) | EP3509614A4 (https=) |
| JP (1) | JP2019535645A (https=) |
| CN (1) | CN109963576A (https=) |
| AU (1) | AU2017322483A1 (https=) |
| CA (1) | CA3036524A1 (https=) |
| WO (1) | WO2018049237A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6178785B2 (ja) | 2011-04-01 | 2017-08-09 | イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔｙ | 細胞浸透性抗ｄｎａ抗体およびｄｎａ修復を阻害するためのその使用 |
| 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 |
| EP4017523A4 (en) * | 2019-08-22 | 2024-01-17 | The Regents Of The University Of California | UBE3A FOR THE TREATMENT OF ANGELMAN SYNDROME |
| WO2025018822A1 (ko) | 2023-07-20 | 2025-01-23 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| KR102814185B1 (ko) | 2023-07-20 | 2025-06-12 | 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 |
|---|---|---|---|---|
| AU2010260145B2 (en) | 2009-06-15 | 2015-08-20 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides |
| US20130273026A1 (en) * | 2012-04-12 | 2013-10-17 | Integrative Enzymatics, Inc. | Composition and method for modulating inflammatory molecules with amylase |
| WO2014130722A1 (en) * | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of forbes-cori disease |
| AU2014218854B2 (en) * | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| AU2015204446A1 (en) * | 2014-01-13 | 2016-07-14 | 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 |
| CA2995673A1 (en) * | 2014-08-27 | 2016-03-03 | Valerion Therapeutics, Llc | Internalizing moieties for treatment of cancer |
-
2017
- 2017-09-08 JP JP2019513898A patent/JP2019535645A/ja active Pending
- 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 US US16/332,331 patent/US10781433B2/en active Active
- 2017-09-08 EP EP17849664.2A patent/EP3509614A4/en not_active Withdrawn
- 2017-09-08 CA CA3036524A patent/CA3036524A1/en not_active Abandoned
- 2017-09-08 WO PCT/US2017/050787 patent/WO2018049237A1/en not_active Ceased
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