JP2019536484A5 - - Google Patents
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- JP2019536484A5 JP2019536484A5 JP2019551256A JP2019551256A JP2019536484A5 JP 2019536484 A5 JP2019536484 A5 JP 2019536484A5 JP 2019551256 A JP2019551256 A JP 2019551256A JP 2019551256 A JP2019551256 A JP 2019551256A JP 2019536484 A5 JP2019536484 A5 JP 2019536484A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- polynucleotide
- ltr
- virus
- hiv
- 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
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- 108091033319 polynucleotide Proteins 0.000 claims description 66
- 102000040430 polynucleotide Human genes 0.000 claims description 66
- 239000002157 polynucleotide Substances 0.000 claims description 66
- 241000700605 Viruses Species 0.000 claims description 18
- 102000004627 Iduronidase Human genes 0.000 claims description 17
- 108010003381 Iduronidase Proteins 0.000 claims description 17
- 229920001184 polypeptide Polymers 0.000 claims description 15
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 15
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 15
- 210000004962 mammalian cell Anatomy 0.000 claims description 13
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims description 12
- 210000004027 cell Anatomy 0.000 claims description 12
- 239000000203 mixture Substances 0.000 claims description 7
- 241000701022 Cytomegalovirus Species 0.000 claims description 6
- 241000725303 Human immunodeficiency virus Species 0.000 claims description 6
- 102100022338 Integrin alpha-M Human genes 0.000 claims description 6
- 238000012217 deletion Methods 0.000 claims description 6
- 230000037430 deletion Effects 0.000 claims description 6
- 238000004806 packaging method and process Methods 0.000 claims description 6
- 241001430294 unidentified retrovirus Species 0.000 claims description 6
- 241000713666 Lentivirus Species 0.000 claims description 5
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims description 4
- 102100039196 CX3C chemokine receptor 1 Human genes 0.000 claims description 4
- 241000713800 Feline immunodeficiency virus Species 0.000 claims description 4
- 101000746022 Homo sapiens CX3C chemokine receptor 1 Proteins 0.000 claims description 4
- 101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 claims description 4
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims description 4
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims description 4
- 201000002883 Scheie syndrome Diseases 0.000 claims description 4
- 102100037029 Transmembrane protein 119 Human genes 0.000 claims description 4
- 101710170979 Transmembrane protein 119 Proteins 0.000 claims description 4
- 239000012634 fragment Substances 0.000 claims description 4
- 230000008488 polyadenylation Effects 0.000 claims description 4
- 210000000130 stem cell Anatomy 0.000 claims description 4
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims description 3
- 241000713340 Human immunodeficiency virus 2 Species 0.000 claims description 3
- 206010039491 Sarcoma Diseases 0.000 claims description 3
- 239000008194 pharmaceutical composition Substances 0.000 claims description 3
- 108010017009 CD11b Antigen Proteins 0.000 claims description 2
- 241000283707 Capra Species 0.000 claims description 2
- 241000713756 Caprine arthritis encephalitis virus Species 0.000 claims description 2
- 108020004705 Codon Proteins 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 241000713730 Equine infectious anemia virus Species 0.000 claims description 2
- 108091006027 G proteins Proteins 0.000 claims description 2
- 102000030782 GTP binding Human genes 0.000 claims description 2
- 108091000058 GTP-Binding Proteins 0.000 claims description 2
- 208000031886 HIV Infections Diseases 0.000 claims description 2
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 108010020382 Hepatocyte Nuclear Factor 1-alpha Proteins 0.000 claims description 2
- 102100022057 Hepatocyte nuclear factor 1-alpha Human genes 0.000 claims description 2
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 2
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 claims description 2
- 101000740205 Homo sapiens Sal-like protein 1 Proteins 0.000 claims description 2
- 102100025136 Macrosialin Human genes 0.000 claims description 2
- 108010057466 NF-kappa B Proteins 0.000 claims description 2
- 102000003945 NF-kappa B Human genes 0.000 claims description 2
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 2
- 102100037204 Sal-like protein 1 Human genes 0.000 claims description 2
- 108700026226 TATA Box Proteins 0.000 claims description 2
- 208000010094 Visna Diseases 0.000 claims description 2
- 208000007502 anemia Diseases 0.000 claims description 2
- 206010003246 arthritis Diseases 0.000 claims description 2
- 108010006025 bovine growth hormone Proteins 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 206010014599 encephalitis Diseases 0.000 claims description 2
- 239000003623 enhancer Substances 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 230000004048 modification Effects 0.000 claims description 2
- 238000012986 modification Methods 0.000 claims description 2
- 230000002062 proliferating effect Effects 0.000 claims description 2
- 102000005962 receptors Human genes 0.000 claims description 2
- 108020003175 receptors Proteins 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 208000011580 syndromic disease Diseases 0.000 claims description 2
- 230000029812 viral genome replication Effects 0.000 claims description 2
- 230000006490 viral transcription Effects 0.000 claims description 2
- 108091010877 Allograft inflammatory factor 1 Proteins 0.000 claims 2
- 241000713311 Simian immunodeficiency virus Species 0.000 claims 2
- 239000000853 adhesive Substances 0.000 claims 1
- 230000001070 adhesive effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 9
- 208000024891 symptom Diseases 0.000 description 3
- 201000004569 Blindness Diseases 0.000 description 2
- 206010011878 Deafness Diseases 0.000 description 2
- 241000714474 Rous sarcoma virus Species 0.000 description 2
- 238000009825 accumulation Methods 0.000 description 2
- 230000006999 cognitive decline Effects 0.000 description 2
- 208000010877 cognitive disease Diseases 0.000 description 2
- 230000003111 delayed effect Effects 0.000 description 2
- 230000004064 dysfunction Effects 0.000 description 2
- 230000010370 hearing loss Effects 0.000 description 2
- 231100000888 hearing loss Toxicity 0.000 description 2
- 208000016354 hearing loss disease Diseases 0.000 description 2
- 208000019622 heart disease Diseases 0.000 description 2
- 230000008140 language development Effects 0.000 description 2
- 230000013016 learning Effects 0.000 description 2
- 208000023504 respiratory system disease Diseases 0.000 description 2
- KZMAWJRXKGLWGS-UHFFFAOYSA-N 2-chloro-n-[4-(4-methoxyphenyl)-1,3-thiazol-2-yl]-n-(3-methoxypropyl)acetamide Chemical compound S1C(N(C(=O)CCl)CCCOC)=NC(C=2C=CC(OC)=CC=2)=C1 KZMAWJRXKGLWGS-UHFFFAOYSA-N 0.000 description 1
- 108010045403 Calcium-Binding Proteins Proteins 0.000 description 1
- 102000005701 Calcium-Binding Proteins Human genes 0.000 description 1
- 241000282693 Cercopithecidae Species 0.000 description 1
- 101000890626 Homo sapiens Allograft inflammatory factor 1 Proteins 0.000 description 1
- 206010061598 Immunodeficiency Diseases 0.000 description 1
- 208000029462 Immunodeficiency disease Diseases 0.000 description 1
- 230000001464 adherent effect Effects 0.000 description 1
- 230000007813 immunodeficiency Effects 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
- 230000002458 infectious effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430795P | 2016-12-06 | 2016-12-06 | |
| US62/430,795 | 2016-12-06 | ||
| PCT/US2017/064913 WO2018106807A1 (en) | 2016-12-06 | 2017-12-06 | Gene therapy for mucopolysaccharidosis, type i |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019536484A JP2019536484A (ja) | 2019-12-19 |
| JP2019536484A5 true JP2019536484A5 (enExample) | 2021-01-21 |
Family
ID=62491308
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019551256A Pending JP2019536484A (ja) | 2016-12-06 | 2017-12-06 | ムコ多糖症ｉ型のための遺伝子治療 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220339296A1 (enExample) |
| EP (1) | EP3551750A4 (enExample) |
| JP (1) | JP2019536484A (enExample) |
| KR (1) | KR20190089988A (enExample) |
| CN (1) | CN110214182A (enExample) |
| AU (1) | AU2017370662A1 (enExample) |
| BR (1) | BR112019011635A2 (enExample) |
| CA (1) | CA3046079A1 (enExample) |
| IL (1) | IL267057A (enExample) |
| MA (1) | MA47847A (enExample) |
| MX (1) | MX2019006552A (enExample) |
| RU (1) | RU2019120721A (enExample) |
| WO (1) | WO2018106807A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| JP7576754B2 (ja) | 2018-04-11 | 2024-11-01 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | 幹細胞移植のための組成物および方法 |
| MX2021001292A (es) * | 2018-07-30 | 2021-07-15 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Metodos para la modificacion genica de celulas hematopoyeticas. |
| CN115109790A (zh) * | 2021-03-23 | 2022-09-27 | 北京据德医药科技有限公司 | 一种重组a-L-艾杜糖醛酸普酶及其制备方法 |
| CA3222751A1 (en) * | 2021-06-08 | 2022-12-15 | Touchlight IP Limited | Lentiviral vector |
| WO2024201066A1 (en) * | 2023-03-31 | 2024-10-03 | Dawn Therapeutics Limited | Novel vector |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2872170B1 (fr) * | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| US20110294114A1 (en) * | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| AU2012267512B2 (en) * | 2011-06-10 | 2014-11-20 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014201252A2 (en) * | 2013-06-13 | 2014-12-18 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
| EP3413896B1 (en) * | 2016-02-12 | 2021-03-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
-
2017
- 2017-12-06 RU RU2019120721A patent/RU2019120721A/ru not_active Application Discontinuation
- 2017-12-06 CA CA3046079A patent/CA3046079A1/en not_active Abandoned
- 2017-12-06 CN CN201780083351.5A patent/CN110214182A/zh active Pending
- 2017-12-06 MA MA047847A patent/MA47847A/fr unknown
- 2017-12-06 AU AU2017370662A patent/AU2017370662A1/en not_active Abandoned
- 2017-12-06 KR KR1020197019344A patent/KR20190089988A/ko not_active Ceased
- 2017-12-06 WO PCT/US2017/064913 patent/WO2018106807A1/en not_active Ceased
- 2017-12-06 JP JP2019551256A patent/JP2019536484A/ja active Pending
- 2017-12-06 EP EP17877511.0A patent/EP3551750A4/en not_active Withdrawn
- 2017-12-06 BR BR112019011635-4A patent/BR112019011635A2/pt not_active IP Right Cessation
- 2017-12-06 US US16/466,130 patent/US20220339296A1/en not_active Abandoned
- 2017-12-06 MX MX2019006552A patent/MX2019006552A/es unknown
-
2019
- 2019-06-03 IL IL267057A patent/IL267057A/en unknown
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