JP2012525141A5 - - Google Patents
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- Publication number
- JP2012525141A5 JP2012525141A5 JP2012507847A JP2012507847A JP2012525141A5 JP 2012525141 A5 JP2012525141 A5 JP 2012525141A5 JP 2012507847 A JP2012507847 A JP 2012507847A JP 2012507847 A JP2012507847 A JP 2012507847A JP 2012525141 A5 JP2012525141 A5 JP 2012525141A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- mirna
- gene
- gene vector
- expression
- 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.)
- Granted
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- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 82
- 239000013598 vector Substances 0.000 claims description 72
- 108090000623 proteins and genes Proteins 0.000 claims description 63
- 108091070501 miRNA Proteins 0.000 claims description 52
- 210000004027 cell Anatomy 0.000 claims description 50
- 239000002679 microRNA Substances 0.000 claims description 49
- 239000002245 particle Substances 0.000 claims description 40
- 239000002773 nucleotide Substances 0.000 claims description 22
- 125000003729 nucleotide group Chemical group 0.000 claims description 22
- 239000008194 pharmaceutical composition Substances 0.000 claims description 21
- 108700019146 Transgenes Proteins 0.000 claims description 20
- 239000003814 drug Substances 0.000 claims description 20
- 238000001415 gene therapy Methods 0.000 claims description 18
- 238000000034 method Methods 0.000 claims description 16
- 238000004519 manufacturing process Methods 0.000 claims description 15
- 108091028066 Mir-126 Proteins 0.000 claims description 12
- 239000013603 viral vector Substances 0.000 claims description 11
- 230000004069 differentiation Effects 0.000 claims description 10
- 108091062140 Mir-223 Proteins 0.000 claims description 9
- 208000002491 severe combined immunodeficiency Diseases 0.000 claims description 8
- 102100025621 Cytochrome b-245 heavy chain Human genes 0.000 claims description 6
- 108090000790 Enzymes Proteins 0.000 claims description 6
- 102000004190 Enzymes Human genes 0.000 claims description 6
- 206010028980 Neoplasm Diseases 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 206010003694 Atrophy Diseases 0.000 claims description 4
- 230000006907 apoptotic process Effects 0.000 claims description 4
- 230000037444 atrophy Effects 0.000 claims description 4
- 208000016532 chronic granulomatous disease Diseases 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- 238000012544 monitoring process Methods 0.000 claims description 4
- 230000004083 survival effect Effects 0.000 claims description 4
- 210000004885 white matter Anatomy 0.000 claims description 4
- 230000000694 effects Effects 0.000 claims description 3
- 108010042681 Galactosylceramidase Proteins 0.000 claims description 2
- 101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 claims description 2
- 102100022338 Integrin alpha-M Human genes 0.000 claims description 2
- 102000006992 Interferon-alpha Human genes 0.000 claims description 2
- 108010047761 Interferon-alpha Proteins 0.000 claims description 2
- 241000713666 Lentivirus Species 0.000 claims description 2
- 108010082739 NADPH Oxidase 2 Proteins 0.000 claims description 2
- 238000002659 cell therapy Methods 0.000 claims description 2
- 229940079593 drug Drugs 0.000 claims description 2
- 230000002132 lysosomal effect Effects 0.000 claims description 2
- 238000009168 stem cell therapy Methods 0.000 claims description 2
- 238000009580 stem-cell therapy Methods 0.000 claims description 2
- 210000001519 tissue Anatomy 0.000 claims 2
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 description 3
- 102000004169 proteins and genes Human genes 0.000 description 2
- 108020005544 Antisense RNA Proteins 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 102000019034 Chemokines Human genes 0.000 description 1
- 108010012236 Chemokines Proteins 0.000 description 1
- 102000004127 Cytokines Human genes 0.000 description 1
- 108090000695 Cytokines Proteins 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 108010052285 Membrane Proteins Proteins 0.000 description 1
- 208000012902 Nervous system disease Diseases 0.000 description 1
- 208000025966 Neurological disease Diseases 0.000 description 1
- 208000006011 Stroke Diseases 0.000 description 1
- 102000001742 Tumor Suppressor Proteins Human genes 0.000 description 1
- 108010040002 Tumor Suppressor Proteins Proteins 0.000 description 1
- 208000036142 Viral infection Diseases 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 230000003213 activating effect Effects 0.000 description 1
- 230000000840 anti-viral effect Effects 0.000 description 1
- 206010003246 arthritis Diseases 0.000 description 1
- -1 c-Fes Proteins 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 239000003184 complementary RNA Substances 0.000 description 1
- 230000006866 deterioration Effects 0.000 description 1
- 108020001507 fusion proteins Proteins 0.000 description 1
- 102000037865 fusion proteins Human genes 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- 208000019622 heart disease Diseases 0.000 description 1
- 239000005556 hormone Substances 0.000 description 1
- 229940088597 hormone Drugs 0.000 description 1
- 230000002519 immonomodulatory effect Effects 0.000 description 1
- 208000026278 immune system disease Diseases 0.000 description 1
- 239000004615 ingredient Substances 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 229940002612 prodrug Drugs 0.000 description 1
- 239000000651 prodrug Substances 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 239000000225 tumor suppressor protein Substances 0.000 description 1
- 230000002227 vasoactive effect Effects 0.000 description 1
- 230000009385 viral infection Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17412409P | 2009-04-30 | 2009-04-30 | |
| US61/174,124 | 2009-04-30 | ||
| PCT/IB2010/001166 WO2010125471A2 (en) | 2009-04-30 | 2010-04-30 | Gene vector |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015175986A Division JP6370278B2 (ja) | 2009-04-30 | 2015-09-07 | 遺伝子ベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012525141A JP2012525141A (ja) | 2012-10-22 |
| JP2012525141A5 true JP2012525141A5 (cg-RX-API-DMAC7.html) | 2013-11-14 |
| JP6144911B2 JP6144911B2 (ja) | 2017-06-07 |
Family
ID=42790671
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012507847A Active JP6144911B2 (ja) | 2009-04-30 | 2010-04-30 | 遺伝子ベクター |
| JP2015175986A Active JP6370278B2 (ja) | 2009-04-30 | 2015-09-07 | 遺伝子ベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015175986A Active JP6370278B2 (ja) | 2009-04-30 | 2015-09-07 | 遺伝子ベクター |
Country Status (23)
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2011011508A (es) | 2009-04-30 | 2012-02-13 | San Raffaele Centro Fond | Vector genico. |
| SI2772265T1 (en) | 2010-05-14 | 2018-06-29 | Oregon Health & Science University | HCMV AND RHCMV RECOMBINANT VECTORS AND THEIR APPLICATIONS |
| WO2012006577A2 (en) * | 2010-07-08 | 2012-01-12 | Duke University | Direct reprogramming of cells to cardiac myocyte fate |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| WO2013152230A1 (en) * | 2012-04-04 | 2013-10-10 | The Trustees Of Columbia University In The City Of New York | Smooth muscle specific inhibition for anti-restenotic therapy |
| GB2537000C (en) | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| CA2878037C (en) * | 2012-07-11 | 2021-08-31 | Sangamo Biosciences, Inc. | Methods and compositions for delivery of biologics |
| CA2891347C (en) | 2012-12-06 | 2018-02-27 | Sigma-Aldrich Co. Llc | Crispr-based genome modification and regulation |
| PL2956477T5 (pl) | 2013-02-15 | 2024-05-27 | Bioverativ Therapeutics Inc. | Zoptymalizowany gen czynnika viii |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| EP3060670B1 (en) | 2013-10-24 | 2019-07-10 | Ospedale San Raffaele S.r.l. | Method |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| EP3318630B1 (en) * | 2015-04-22 | 2020-11-04 | iPS Academia Japan, Inc. | Method for sorting tissue cells |
| WO2016201507A1 (en) * | 2015-06-15 | 2016-12-22 | Murdoch Childrens Research Institute | Method of measuring chimerism |
| US10849920B2 (en) | 2015-10-05 | 2020-12-01 | Modernatx, Inc. | Methods for therapeutic administration of messenger ribonucleic acid drugs |
| US10188750B1 (en) | 2015-10-23 | 2019-01-29 | University Of South Florida | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| RS63548B1 (sr) | 2016-02-01 | 2022-09-30 | Bioverativ Therapeutics Inc | Optimizovani geni faktora viii |
| GB201612214D0 (en) | 2016-07-14 | 2016-08-31 | Univ Oxford Innovation Ltd | Method |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| CA3040303A1 (en) * | 2016-10-18 | 2018-04-26 | Oregon Health & Science University | Cytomegalovirus vectors eliciting t cells restricted by major histocompatibility complex e molecules |
| CN106566838B (zh) * | 2016-11-14 | 2019-11-01 | 上海伯豪生物技术有限公司 | 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用 |
| WO2018193119A1 (en) * | 2017-04-21 | 2018-10-25 | Ospedale San Raffaele S.R.L. | Gene therapy |
| IT201700086403A1 (it) * | 2017-07-27 | 2019-01-27 | Univ Del Piemonte Orientale | Endothelial-specific promoter sequences and uses thereof |
| KR20250040746A (ko) | 2017-08-09 | 2025-03-24 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
| CN111918674A (zh) | 2018-02-01 | 2020-11-10 | 比奥维拉迪维治疗股份有限公司 | 表达因子viii的慢病毒载体的用途 |
| JP7602454B2 (ja) | 2018-08-09 | 2024-12-18 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
| SG11202105880TA (en) | 2018-12-06 | 2021-07-29 | Bioverativ Therapeutics Inc | Use of lentiviral vectors expressing factor ix |
| US12403164B2 (en) | 2019-09-30 | 2025-09-02 | Bioverativ Therapeutics Inc. | Lentiviral vector formulations |
| CA3161175A1 (en) * | 2019-11-12 | 2021-05-20 | The Regents Of The University Of California | Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease |
| BR112022026127A2 (pt) | 2020-06-24 | 2023-01-17 | Bioverativ Therapeutics Inc | Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína |
| AU2021330936A1 (en) | 2020-08-23 | 2023-05-04 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| JP2024534123A (ja) | 2021-08-23 | 2024-09-18 | バイオベラティブ セラピューティクス インコーポレイテッド | バキュロウイルス発現システム |
| TW202323274A (zh) | 2021-08-23 | 2023-06-16 | 美商百歐維拉提夫治療公司 | 優化因子viii基因 |
| CN115725656A (zh) * | 2021-09-01 | 2023-03-03 | 联邦生物科技(珠海横琴)有限公司 | 一种以细胞质rna病毒为载体递送干扰rna的方法 |
| AU2022355088A1 (en) | 2021-09-30 | 2024-05-16 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| EP4561638A2 (en) * | 2022-07-25 | 2025-06-04 | ModernaTX, Inc. | Engineered polynucleotides for cell selective expression |
| WO2025122739A1 (en) * | 2023-12-05 | 2025-06-12 | University Of Pittsburgh-Of The Commonwealth System Of Higher Education | Compositions and methods for treating a heart disease |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5723315A (en) | 1996-08-23 | 1998-03-03 | Genetics Institute, Inc. | Secreted proteins and polynucleotides encoding them |
| US6118001A (en) | 1996-08-07 | 2000-09-12 | Darwin Discovery, Ltd. | Hydroxamic and carboxylic acid derivatives having MMP and TNF inhibitory activity |
| US6126939A (en) | 1996-09-03 | 2000-10-03 | Yeda Research And Development Co. Ltd. | Anti-inflammatory dipeptide and pharmaceutical composition thereof |
| NZ532060A (en) * | 2001-10-02 | 2005-11-25 | Inst Clayton De La Rech | Methods and compositions relating to restricted expression lentiviral vectors and their applications |
| EP2290071B1 (en) | 2004-05-28 | 2014-12-31 | Asuragen, Inc. | Methods and compositions involving microRNA |
| US20060134784A1 (en) * | 2004-11-30 | 2006-06-22 | Basch Ross S | Methods and compositions for the growth and maintenance of stem cells |
| SI2002003T1 (sl) | 2005-05-27 | 2016-05-31 | Ospedale San Raffaele S.R.L. | Genski vektor, ki vsebuje mi-RNA |
| CA2610702A1 (en) * | 2005-06-03 | 2006-12-07 | Michael Zenon Michael | Targeting cells with altered microrna expression |
| US20090180989A1 (en) * | 2005-10-05 | 2009-07-16 | Synageva Biopharma Corp. | Compositions and methods for delivering nucleotide sequences to vertebrates |
| EP2479286B1 (en) | 2006-01-05 | 2013-11-13 | The Ohio State University Research Foundation | MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers |
| US20080095750A1 (en) | 2006-05-10 | 2008-04-24 | Pennington Biomedical Research Center, Louisiana State University System | Use of adipose-derived stem cells for treatment of leukodystrophies |
| WO2008073921A2 (en) | 2006-12-08 | 2008-06-19 | Asuragen, Inc. | Mir-126 regulated genes and pathways as targets for therapeutic intervention |
| ES2635726T3 (es) | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| EP2364362B1 (en) | 2008-11-12 | 2015-10-21 | Ospedale San Raffaele S.r.l. | Gene vector for inducing transgene-specific immune tolerance |
| MX2011011508A (es) * | 2009-04-30 | 2012-02-13 | San Raffaele Centro Fond | Vector genico. |
-
2010
- 2010-04-30 MX MX2011011508A patent/MX2011011508A/es active IP Right Grant
- 2010-04-30 PT PT107233058T patent/PT2424571T/pt unknown
- 2010-04-30 EP EP20167404.1A patent/EP3733214A1/en active Pending
- 2010-04-30 JP JP2012507847A patent/JP6144911B2/ja active Active
- 2010-04-30 SI SI201032001T patent/SI2424571T1/sl unknown
- 2010-04-30 PL PL10723305T patent/PL2424571T3/pl unknown
- 2010-04-30 EA EA201171335A patent/EA023938B1/ru not_active IP Right Cessation
- 2010-04-30 HR HRP20200647TT patent/HRP20200647T1/hr unknown
- 2010-04-30 SG SG2011080009A patent/SG175839A1/en unknown
- 2010-04-30 AU AU2010243276A patent/AU2010243276B2/en active Active
- 2010-04-30 LT LTEP10723305.8T patent/LT2424571T/lt unknown
- 2010-04-30 SG SG10201610827RA patent/SG10201610827RA/en unknown
- 2010-04-30 KR KR1020117028569A patent/KR101793615B1/ko active Active
- 2010-04-30 DK DK10723305.8T patent/DK2424571T3/da active
- 2010-04-30 HU HUE10723305A patent/HUE049845T2/hu unknown
- 2010-04-30 CN CN201080030337.7A patent/CN102596255B/zh active Active
- 2010-04-30 ES ES10723305T patent/ES2786039T3/es active Active
- 2010-04-30 US US13/266,381 patent/US10287579B2/en active Active
- 2010-04-30 CA CA2759438A patent/CA2759438C/en active Active
- 2010-04-30 WO PCT/IB2010/001166 patent/WO2010125471A2/en not_active Ceased
- 2010-04-30 BR BRPI1010873A patent/BRPI1010873B1/pt active IP Right Grant
- 2010-04-30 SM SM20200224T patent/SMT202000224T1/it unknown
- 2010-04-30 EP EP10723305.8A patent/EP2424571B1/en active Active
-
2011
- 2011-10-23 IL IL215804A patent/IL215804A/en active IP Right Grant
-
2015
- 2015-09-07 JP JP2015175986A patent/JP6370278B2/ja active Active
-
2017
- 2017-08-31 US US15/693,112 patent/US9951328B2/en active Active
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2019
- 2019-04-15 US US16/384,571 patent/US11407996B2/en active Active
-
2020
- 2020-05-04 CY CY20201100405T patent/CY1123218T1/el unknown
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2022
- 2022-06-30 US US17/855,135 patent/US20220333103A1/en active Pending
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