EA023938B1 - Вектор гена - Google Patents
Вектор гена Download PDFInfo
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- EA023938B1 EA023938B1 EA201171335A EA201171335A EA023938B1 EA 023938 B1 EA023938 B1 EA 023938B1 EA 201171335 A EA201171335 A EA 201171335A EA 201171335 A EA201171335 A EA 201171335A EA 023938 B1 EA023938 B1 EA 023938B1
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- cell
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- mirna
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| US10266850B2 (en) | 2012-05-25 | 2019-04-23 | The Regents Of The University Of California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| JP6329537B2 (ja) | 2012-07-11 | 2018-05-23 | サンガモ セラピューティクス， インコーポレイテッド | 生物学的薬剤の送達のための方法および組成物 |
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| DK3620534T3 (da) | 2013-03-14 | 2021-12-06 | Caribou Biosciences Inc | Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer |
| CA2927712A1 (en) | 2013-10-24 | 2015-04-30 | Ospedale San Raffaele S.R.L. | Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| US10501811B2 (en) | 2015-04-22 | 2019-12-10 | Kyoto University | Method for sorting tissue cells |
| WO2016201507A1 (en) * | 2015-06-15 | 2016-12-22 | Murdoch Childrens Research Institute | Method of measuring chimerism |
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| US10188750B1 (en) | 2015-10-23 | 2019-01-29 | University Of South Florida | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| PH12018501628B1 (en) | 2016-02-01 | 2023-11-10 | Bioverativ Therapeutics Inc | Optimized factor viii genes |
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| CR20190205A (es) * | 2016-10-18 | 2020-01-07 | Univ Oregon Health & Science | Vectores de citomegalovirus que provocan células t restringidas por moléculas del complejo mayor de histocompatibilidad e |
| 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 |
| EP4491233A3 (en) * | 2017-04-21 | 2025-04-16 | 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 |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| JP2021512126A (ja) | 2018-02-01 | 2021-05-13 | 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 | 第ｖｉｉｉ因子を発現するレンチウイルスベクターの使用 |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CN113396223A (zh) | 2018-12-06 | 2021-09-14 | 比奥维拉迪维治疗股份有限公司 | 表达因子ix的慢病毒载体的用途 |
| CN115279896A (zh) | 2019-09-30 | 2022-11-01 | 比奥维拉迪维治疗股份有限公司 | 慢病毒载体配制品 |
| US20220378937A1 (en) * | 2019-11-12 | 2022-12-01 | The Regents Of The University Of California | Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease |
| IL299333A (en) | 2020-06-24 | 2023-02-01 | Bioverativ Therapeutics Inc | Methods for the purification of viral vectors |
| CN116194152A (zh) | 2020-08-23 | 2023-05-30 | 比奥维拉迪维治疗股份有限公司 | 用于改进封闭端DNA（ceDNA）的产生的经修饰的杆状病毒系统 |
| EP4392566A2 (en) | 2021-08-23 | 2024-07-03 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
| AU2022332276A1 (en) | 2021-08-23 | 2024-04-04 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| WO2023056331A1 (en) | 2021-09-30 | 2023-04-06 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| MA71632A (fr) * | 2022-07-25 | 2025-05-30 | Modernatx, Inc. | Polynucléotides modifiés pour l'expression sélective de cellules |
| 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 |
Citations (1)
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|---|---|---|---|---|
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Family Cites Families (14)
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|---|---|---|---|---|
| US5723315A (en) | 1996-08-23 | 1998-03-03 | Genetics Institute, Inc. | Secreted proteins and polynucleotides encoding them |
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| EP2474616B1 (en) | 2004-05-28 | 2015-07-08 | Asuragen, Inc. | Methods and compositions involving microRNA |
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| KR20130114758A (ko) * | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Ｍｉ-ｒｎａ를 포함하는 유전자 벡터 |
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| CA2671296A1 (en) | 2006-12-08 | 2008-06-19 | Asuragen, Inc. | Mir-126 regulated genes and pathways as targets for therapeutic intervention |
| EP3252161B1 (en) | 2007-06-06 | 2021-11-17 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
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Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
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Non-Patent Citations (8)
| Title |
|---|
| BROWN BRIAN D. ET AL.: "Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state". NATURE BIOTECHNOLOGY, vol. 25, no. 12, 16 November 2007 (2007-11-16), pages 1457-1467, XP002471752, ISSN: 1087-0156, cited in the application, the whole document * |
| CARTIER NATHALIE ET AL.: "Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy". CURRENT OPINION IN MOLECULAR THERAPEUTICS, vol. 10, no. 5, October 2008 (2008-10), pages 471-478, XP008127749, ISSN: 1464-8431, the whole document * |
| GARZON RAMIRO ET AL.: "MicroRNA fingerprints during human megakaryocytopoiesis". PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES, vol. 103, no. 13, 28 March 2006 (2006-03-28), pages 5078-5083, XP002465978, ISSN: 0027-8424, the whole document * |
| GENTNER BERNHARD ET AL.: "Characterization of Hematopoietic Stem Cell Specific microRNA: From Physiologic Function to Gene Therapy Applications". MOLECULAR THERAPY, vol. 17, no. Suppl. 1, May 2009 (2009-05), page S20, XP008127618, ISSN: 1525-0016, the whole document & 12TH ANNUAL MEETING OF THE AMERICAN SOCIETY OF GENE THERAPY; SAN DIEGO, CA, USA; MAY 27-30, 2009 * |
| GENTNER ET AL.: "Exploiting microRNA expression profiles for lineage- and differentiation state-specific transgene expression in hematopoietic, neural and embryonic stem cells". BLOOD CELLS, MOLECULES AND DISEASES, vol. 40, no. 2, 12 February 2008 (2008-02-12), page 267, XP022477840, ISSN: 1079-9796, the whole document * |
| LECHMAN ERIC R. ET AL.: "High Levels of MicroRNA-126 Bioactivity Specify the LSC Compartment in AML" BLOOD, vol. 112, no. 11, November 2008 (2008-11), page 193, XP008127737, ISSN: 0006-4971, the whole document & 50TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; SAN FRANCISCO, CA, USA; DECEMBER 06-09, 2008 * |
| PAPAPETROU EIRINI P. ET AL.: "MicroRNA-mediated gene regulation effectively restricts in vivo transgene expression in hematopoietic stem cell progeny", BLOOD, vol. 110, no. 11, Part 1, November 2007 (2007-11), pages 64A-65A, XP008127620, ISSN: 0006-4971, the whole document & 49TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY; ATLANTA, GA, USA; DECEMBER 08-11, 2007 * |
| RYSER MARTIN F. ET AL.: "Gene therapy for chronic granulomatous disease". EXPERT OPINION ON BIOLOGICAL THERAPY, vol. 7, no. 12, December 2007 (2007-12), pages 1799-1809, XP008127764, ISSN: 1744-7682, the whole document * |
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