JPWO2021221995A5 - - Google Patents
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- Publication number
- JPWO2021221995A5 JPWO2021221995A5 JP2022566010A JP2022566010A JPWO2021221995A5 JP WO2021221995 A5 JPWO2021221995 A5 JP WO2021221995A5 JP 2022566010 A JP2022566010 A JP 2022566010A JP 2022566010 A JP2022566010 A JP 2022566010A JP WO2021221995 A5 JPWO2021221995 A5 JP WO2021221995A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- acid sequence
- vector
- capsid
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims 28
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 18
- 239000013598 vector Substances 0.000 claims 15
- 210000000234 capsid Anatomy 0.000 claims 11
- 239000013608 rAAV vector Substances 0.000 claims 11
- 108020004707 nucleic acids Proteins 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 10
- 101710108545 Viral protein 1 Proteins 0.000 claims 8
- 108700019146 Transgenes Proteins 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 6
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 239000003623 enhancer Substances 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 208000022526 Canavan disease Diseases 0.000 claims 3
- 210000004556 brain Anatomy 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000701022 Cytomegalovirus Species 0.000 claims 2
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims 2
- 241000702623 Minute virus of mice Species 0.000 claims 2
- 108010006025 bovine growth hormone Proteins 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- 239000005090 green fluorescent protein Substances 0.000 claims 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims 2
- 239000003550 marker Substances 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 241000425548 Adeno-associated virus 3A Species 0.000 claims 1
- 241000958487 Adeno-associated virus 3B Species 0.000 claims 1
- 108700042658 GAP-43 Proteins 0.000 claims 1
- 241000287828 Gallus gallus Species 0.000 claims 1
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 210000005013 brain tissue Anatomy 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 238000003384 imaging method Methods 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 238000011002 quantification Methods 0.000 claims 1
- 238000009877 rendering Methods 0.000 claims 1
- 238000004114 suspension culture Methods 0.000 claims 1
- 230000026683 transduction Effects 0.000 claims 1
- 238000010361 transduction Methods 0.000 claims 1
- 230000010415 tropism Effects 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063016507P | 2020-04-28 | 2020-04-28 | |
| US63/016,507 | 2020-04-28 | ||
| US202063077144P | 2020-09-11 | 2020-09-11 | |
| US63/077,144 | 2020-09-11 | ||
| PCT/US2021/028658 WO2021221995A1 (en) | 2020-04-28 | 2021-04-22 | Modified nucleic acids encoding aspartoacylase (aspa) and vector for gene therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023524024A JP2023524024A (ja) | 2023-06-08 |
| JPWO2021221995A5 true JPWO2021221995A5 (zh) | 2024-05-01 |
Family
ID=78373856
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022566010A Pending JP2023524024A (ja) | 2020-04-28 | 2021-04-22 | アスパルトアシラーゼ（ａｓｐａ）をコードする修飾核酸及び遺伝子治療のためのベクター |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230165977A1 (zh) |
| EP (1) | EP4142759A4 (zh) |
| JP (1) | JP2023524024A (zh) |
| KR (1) | KR20230003012A (zh) |
| CN (1) | CN115461066A (zh) |
| AU (1) | AU2021263534A1 (zh) |
| BR (1) | BR112022021964A2 (zh) |
| CA (1) | CA3174070A1 (zh) |
| IL (1) | IL297605A (zh) |
| TW (1) | TW202206599A (zh) |
| WO (1) | WO2021221995A1 (zh) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3234809A1 (en) | 2021-10-20 | 2023-04-27 | Steven Goldman | Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss |
| WO2023081633A1 (en) | 2021-11-02 | 2023-05-11 | University Of Rochester | Tcf7l2 mediated remyelination in the brain |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3002982A1 (en) * | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| US10610606B2 (en) * | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
-
2021
- 2021-04-22 BR BR112022021964A patent/BR112022021964A2/pt unknown
- 2021-04-22 CN CN202180031917.6A patent/CN115461066A/zh active Pending
- 2021-04-22 KR KR1020227041079A patent/KR20230003012A/ko unknown
- 2021-04-22 CA CA3174070A patent/CA3174070A1/en active Pending
- 2021-04-22 JP JP2022566010A patent/JP2023524024A/ja active Pending
- 2021-04-22 US US17/997,312 patent/US20230165977A1/en active Pending
- 2021-04-22 WO PCT/US2021/028658 patent/WO2021221995A1/en unknown
- 2021-04-22 EP EP21795587.1A patent/EP4142759A4/en active Pending
- 2021-04-22 AU AU2021263534A patent/AU2021263534A1/en active Pending
- 2021-04-28 TW TW110115439A patent/TW202206599A/zh unknown
-
2022
- 2022-10-24 IL IL297605A patent/IL297605A/en unknown
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