JP2018531614A5 - - Google Patents
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- JP2018531614A5 JP2018531614A5 JP2018521965A JP2018521965A JP2018531614A5 JP 2018531614 A5 JP2018531614 A5 JP 2018531614A5 JP 2018521965 A JP2018521965 A JP 2018521965A JP 2018521965 A JP2018521965 A JP 2018521965A JP 2018531614 A5 JP2018531614 A5 JP 2018531614A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide expression
- expression construct
- sequence
- promoter
- transgene
- 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.)
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- 108091033319 polynucleotide Proteins 0.000 claims description 43
- 239000002157 polynucleotide Substances 0.000 claims description 43
- 102000040430 polynucleotide Human genes 0.000 claims description 43
- 108700019146 Transgenes Proteins 0.000 claims description 23
- 108010071690 Prealbumin Proteins 0.000 claims description 17
- 102000009190 Transthyretin Human genes 0.000 claims description 17
- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102000004169 proteins and genes Human genes 0.000 claims description 12
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 11
- 239000013607 AAV vector Substances 0.000 claims description 10
- 210000004185 liver Anatomy 0.000 claims description 9
- 101710163270 Nuclease Proteins 0.000 claims description 8
- 101710181917 Serine proteinase inhibitor 1 Proteins 0.000 claims description 8
- 239000003623 enhancer Substances 0.000 claims description 8
- 239000012212 insulator Substances 0.000 claims description 8
- 125000006850 spacer group Chemical group 0.000 claims description 8
- 208000031220 Hemophilia Diseases 0.000 claims description 6
- 208000009292 Hemophilia A Diseases 0.000 claims description 6
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 6
- 230000002950 deficient Effects 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 230000008488 polyadenylation Effects 0.000 claims description 4
- 241000702321 Microvirus Species 0.000 claims description 2
- 241001529936 Murinae Species 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 239000013598 vector Substances 0.000 claims description 2
- 108091033409 CRISPR Proteins 0.000 claims 3
- 238000010354 CRISPR gene editing Methods 0.000 claims 3
- 238000010459 TALEN Methods 0.000 claims 2
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 241000702623 Minute virus of mice Species 0.000 claims 1
- 238000000034 method Methods 0.000 description 7
- 241000124008 Mammalia Species 0.000 description 4
- 108010088751 Albumins Proteins 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 1
- 210000003719 b-lymphocyte Anatomy 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 239000003112 inhibitor Substances 0.000 description 1
- 238000002493 microarray Methods 0.000 description 1
- 150000003431 steroids Chemical class 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021110561A JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562247469P | 2015-10-28 | 2015-10-28 | |
| US62/247,469 | 2015-10-28 | ||
| US201662307897P | 2016-03-14 | 2016-03-14 | |
| US62/307,897 | 2016-03-14 | ||
| US201662315438P | 2016-03-30 | 2016-03-30 | |
| US62/315,438 | 2016-03-30 | ||
| US201662326229P | 2016-04-22 | 2016-04-22 | |
| US62/326,229 | 2016-04-22 | ||
| US201662355106P | 2016-06-27 | 2016-06-27 | |
| US62/355,106 | 2016-06-27 | ||
| PCT/US2016/042099 WO2017074526A1 (en) | 2015-10-28 | 2016-07-13 | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021110561A Division JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531614A JP2018531614A (ja) | 2018-11-01 |
| JP2018531614A5 true JP2018531614A5 (https=) | 2019-08-15 |
| JP6909212B2 JP6909212B2 (ja) | 2021-07-28 |
Family
ID=58631984
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521965A Active JP6909212B2 (ja) | 2015-10-28 | 2016-07-13 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
| JP2021110561A Active JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021110561A Active JP7324249B2 (ja) | 2015-10-28 | 2021-07-02 | 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (4) | US10143760B2 (https=) |
| EP (1) | EP3368664A4 (https=) |
| JP (2) | JP6909212B2 (https=) |
| KR (2) | KR102766526B1 (https=) |
| CN (2) | CN115141836B (https=) |
| AU (1) | AU2016343887B2 (https=) |
| CA (1) | CA3002524A1 (https=) |
| CO (1) | CO2018005215A2 (https=) |
| IL (1) | IL258826B2 (https=) |
| MX (1) | MX2018005272A (https=) |
| MY (1) | MY189674A (https=) |
| PE (1) | PE20181206A1 (https=) |
| PH (1) | PH12018500927B1 (https=) |
| SA (1) | SA518391456B1 (https=) |
| SG (1) | SG11201803547RA (https=) |
| WO (1) | WO2017074526A1 (https=) |
| ZA (1) | ZA201802723B (https=) |
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