JP2019517281A5 - - Google Patents
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- JP2019517281A5 JP2019517281A5 JP2019517200A JP2019517200A JP2019517281A5 JP 2019517281 A5 JP2019517281 A5 JP 2019517281A5 JP 2019517200 A JP2019517200 A JP 2019517200A JP 2019517200 A JP2019517200 A JP 2019517200A JP 2019517281 A5 JP2019517281 A5 JP 2019517281A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- ltr
- hiv
- mammalian
- hematopoietic stem
- 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 52
- 102000040430 polynucleotide Human genes 0.000 claims description 52
- 239000002157 polynucleotide Substances 0.000 claims description 52
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims description 22
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims description 22
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 22
- 229920001184 polypeptide Polymers 0.000 claims description 19
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 19
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 19
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 12
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 12
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims description 12
- 241000713666 Lentivirus Species 0.000 claims description 11
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims description 11
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 claims description 9
- 241000700605 Viruses Species 0.000 claims description 7
- 239000008194 pharmaceutical composition 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
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims description 4
- 102100039196 CX3C chemokine receptor 1 Human genes 0.000 claims description 4
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- 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
- 241000713340 Human immunodeficiency virus 2 Species 0.000 claims description 4
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims description 4
- 241000714474 Rous sarcoma virus Species 0.000 claims description 4
- 241000713311 Simian immunodeficiency virus Species 0.000 claims description 4
- 102100037029 Transmembrane protein 119 Human genes 0.000 claims description 4
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- 239000012634 fragment Substances 0.000 claims description 4
- 230000008488 polyadenylation Effects 0.000 claims description 4
- 208000024891 symptom Diseases 0.000 claims description 4
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims description 3
- 210000000130 stem cell Anatomy 0.000 claims description 3
- 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 claims description 2
- 102100026439 Adhesion G protein-coupled receptor E1 Human genes 0.000 claims description 2
- 101710096331 Adhesion G protein-coupled receptor E1 Proteins 0.000 claims description 2
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims description 2
- 108010017009 CD11b Antigen Proteins 0.000 claims description 2
- 108010045403 Calcium-Binding Proteins Proteins 0.000 claims description 2
- 102000005701 Calcium-Binding Proteins Human genes 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
- 208000031886 HIV Infections Diseases 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
- 101000890626 Homo sapiens Allograft inflammatory factor 1 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
- 241000713883 Myeloproliferative sarcoma virus Species 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
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims description 2
- 108010077524 Peptide Elongation Factor 1 Proteins 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
- 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
- 230000004048 modification Effects 0.000 claims description 2
- 238000012986 modification Methods 0.000 claims description 2
- 239000013642 negative control Substances 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 230000029812 viral genome replication Effects 0.000 claims description 2
- 230000006490 viral transcription Effects 0.000 claims description 2
- 241000713800 Feline immunodeficiency virus Species 0.000 claims 2
- 208000025014 late infantile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 241000124008 Mammalia Species 0.000 claims 1
- 241000283080 Proboscidea <mammal> Species 0.000 claims 1
- 238000000034 method Methods 0.000 description 9
- 210000004027 cell Anatomy 0.000 description 8
- 210000004962 mammalian cell Anatomy 0.000 description 8
- 201000004569 Blindness Diseases 0.000 description 1
- 206010010904 Convulsion Diseases 0.000 description 1
- 241000282324 Felis Species 0.000 description 1
- 230000006999 cognitive decline Effects 0.000 description 1
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- 230000007423 decrease Effects 0.000 description 1
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662349505P | 2016-06-13 | 2016-06-13 | |
| US62/349,505 | 2016-06-13 | ||
| US201762457498P | 2017-02-10 | 2017-02-10 | |
| US62/457,498 | 2017-02-10 | ||
| PCT/US2017/037230 WO2017218519A1 (en) | 2016-06-13 | 2017-06-13 | Gene therapy of neuronal ceroid lipofuscinoses |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019517281A JP2019517281A (ja) | 2019-06-24 |
| JP2019517281A5 true JP2019517281A5 (enExample) | 2020-07-09 |
Family
ID=60663744
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019517200A Pending JP2019517281A (ja) | 2016-06-13 | 2017-06-13 | 神経セロイドリポフスチン症の遺伝子治療 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220323612A1 (enExample) |
| EP (1) | EP3468594A4 (enExample) |
| JP (1) | JP2019517281A (enExample) |
| CN (1) | CN109475619A (enExample) |
| AU (1) | AU2017283474A1 (enExample) |
| CA (1) | CA3058735A1 (enExample) |
| WO (1) | WO2017218519A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| CN105939767B (zh) | 2014-01-08 | 2018-04-06 | 弗洛设计声能学公司 | 具有双声电泳腔的声电泳装置 |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| KR102673203B1 (ko) | 2016-04-20 | 2024-06-10 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | Pklr의 유전자 발현 증강을 위한 조성물 및 방법 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| AU2018352583B2 (en) | 2017-10-16 | 2025-04-17 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency |
| CN114900773A (zh) | 2017-12-14 | 2022-08-12 | 弗洛设计声能学公司 | 声泳系统及其操作方法、控制声换能器及声学系统的方法 |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| IL280465B2 (en) * | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| EA202192328A1 (ru) * | 2019-03-22 | 2022-02-21 | Раш Юниверсити Медикал Сентер | Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления |
| CN112522321B (zh) * | 2020-12-16 | 2022-04-08 | 北京艺妙神州医药科技有限公司 | 一种基因治疗载体及其用途 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2717922T3 (en) * | 2011-06-10 | 2019-01-07 | Bluebird Bio Inc | GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE |
| WO2014089449A1 (en) * | 2012-12-07 | 2014-06-12 | Rush University Nedical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| WO2016028879A1 (en) * | 2014-08-19 | 2016-02-25 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating immune response |
-
2017
- 2017-06-13 US US16/309,412 patent/US20220323612A1/en not_active Abandoned
- 2017-06-13 AU AU2017283474A patent/AU2017283474A1/en not_active Abandoned
- 2017-06-13 WO PCT/US2017/037230 patent/WO2017218519A1/en not_active Ceased
- 2017-06-13 CN CN201780043899.7A patent/CN109475619A/zh active Pending
- 2017-06-13 JP JP2019517200A patent/JP2019517281A/ja active Pending
- 2017-06-13 CA CA3058735A patent/CA3058735A1/en not_active Abandoned
- 2017-06-13 EP EP17813925.9A patent/EP3468594A4/en not_active Withdrawn
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