JP2013509168A5 - - Google Patents
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- JP2013509168A5 JP2013509168A5 JP2012535849A JP2012535849A JP2013509168A5 JP 2013509168 A5 JP2013509168 A5 JP 2013509168A5 JP 2012535849 A JP2012535849 A JP 2012535849A JP 2012535849 A JP2012535849 A JP 2012535849A JP 2013509168 A5 JP2013509168 A5 JP 2013509168A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- expression cassette
- vector
- seq
- acid 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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- 108020004707 nucleic acids Proteins 0.000 claims 18
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 230000014509 gene expression Effects 0.000 claims 12
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 238000001415 gene therapy Methods 0.000 claims 3
- 210000002064 heart cell Anatomy 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 230000001105 regulatory Effects 0.000 claims 3
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims 2
- 230000001225 therapeutic Effects 0.000 claims 2
- 102000034451 ATPases Human genes 0.000 claims 1
- 108091006096 ATPases Proteins 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 102000004127 Cytokines Human genes 0.000 claims 1
- 108090000695 Cytokines Proteins 0.000 claims 1
- 102100019126 HBB Human genes 0.000 claims 1
- 108091005902 Hemoglobin subunit beta Proteins 0.000 claims 1
- 229920002459 Intron Polymers 0.000 claims 1
- 102000004310 Ion Channels Human genes 0.000 claims 1
- 108090000862 Ion Channels Proteins 0.000 claims 1
- 102100012897 PGF Human genes 0.000 claims 1
- 101710014083 PGF Proteins 0.000 claims 1
- -1 SERCA2a Proteins 0.000 claims 1
- 102100015249 VEGFA Human genes 0.000 claims 1
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 claims 1
- 239000002870 angiogenesis inducing agent Substances 0.000 claims 1
- 230000000295 complement Effects 0.000 claims 1
- 230000002708 enhancing Effects 0.000 claims 1
- 239000003102 growth factor Substances 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000003612 virological Effects 0.000 claims 1
Claims (18)
- 配列番号１、配列番号２、配列番号３、配列番号４、配列番号５、配列番号６、配列番号７、配列番号８、これらの配列のいずれかに対して９５％の同一性を有する配列、およびこれらの機能性断片からなる群から選択される配列を含む、心臓特異的遺伝子発現を増強するための６００ヌクレオチド以下の核酸調節因子。
- 配列番号１、これに対して９５％の同一性を有する配列、またはこれらの機能性断片を含む、請求項１に記載の核酸調節因子。
- 請求項１に記載の調節因子またはこの相補体にストリンジェントな条件下でハイブリダイズする、６００ヌクレオチド以下の核酸調節因子。
- ２５０ヌクレオチド以下である、請求項１〜３のいずれか一項に記載の核酸調節断片。
- プロモーターおよび導入遺伝子に機能的に連結された、請求項１〜４のいずれか一項に記載の核酸調節因子を含む、核酸発現カセット。
- 請求項１〜４のいずれか一項に記載の２つ以上の核酸調節因子を含む、請求項５に記載の核酸発現カセット。
- ２つ以上の調節因子が同一である、請求項６に記載の核酸発現カセット。
- プロモーターが心臓特異的プロモーターである、請求項５〜７のいずれか一項に記載の核酸発現カセット。
- プロモーターがミオシン重鎖遺伝子、特にミオシン重鎖α（αＭＨＣ）遺伝子由来である、請求項８に記載の核酸発現カセット。
- βグロビンイントロンをさらに含む、請求項５〜９のいずれか一項に記載の核酸発現カセット。
- 導入遺伝子が、ＶＥＧＦまたはＰｌＧＦなどの血管形成因子、ＳＥＲＣＡ２ａなどのＡＴＰアーゼ、イオンチャネル、サイトカインおよび成長因子からなる群から選択される治療用タンパク質をコードする、請求項５〜１０のいずれか一項に記載の核酸発現カセット。
- 請求項１〜４のいずれか一項に記載の調節因子を含む、ベクター。
- 請求項５〜１１のいずれか一項に記載の核酸発現カセットを含む、請求項１２に記載のベクター。
- ウイルスベクター、特にレンチウイルスベクターまたはＡＡＶベクターである、請求項１２または１３に記載のベクター。
- ＡＡＶ９ベクターまたはＡＡＶ２／９ベクターである、請求項１４に記載のベクター。
- 遺伝子治療における、請求項１〜３のいずれか一項に記載の核酸調節因子の使用。
- 請求項５〜１１のいずれか一項に記載の核酸発現カセットを心臓細胞内に導入する段階；
導入遺伝子タンパク質産物を心臓細胞内で発現させる段階
を含む、心臓細胞においてタンパク質を発現させるためのインビトロ方法。 - 請求項１１に記載の核酸発現カセット、または請求項１１に記載の核酸発現カセットを含むベクターを被験体の心臓内に導入する段階；
治療量のタンパク質を心臓内で発現させる段階
を含む、遺伝子治療を必要とするヒト以外の被験体に対する遺伝子治療の方法。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP09174519.0 | 2009-10-29 | ||
| EP09174519 | 2009-10-29 | ||
| PCT/EP2010/066474 WO2011051450A1 (en) | 2009-10-29 | 2010-10-29 | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013509168A JP2013509168A (ja) | 2013-03-14 |
| JP2013509168A5 true JP2013509168A5 (ja) | 2013-12-19 |
| JP5823969B2 JP5823969B2 (ja) | 2015-11-25 |
Family
ID=43414855
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012535849A Expired - Fee Related JP5823969B2 (ja) | 2009-10-29 | 2010-10-29 | 心臓特異的核酸調節因子ならびにこの方法および使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9353164B2 (ja) |
| EP (1) | EP2494058B1 (ja) |
| JP (1) | JP5823969B2 (ja) |
| AU (1) | AU2010311376B2 (ja) |
| CA (1) | CA2778945A1 (ja) |
| WO (1) | WO2011051450A1 (ja) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2494058B1 (en) | 2009-10-29 | 2017-04-05 | Vib Vzw | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
| US8859517B2 (en) | 2011-08-08 | 2014-10-14 | Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione | Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT) |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| WO2014077863A1 (en) * | 2012-11-19 | 2014-05-22 | Nature Technology Corporation | Replicative minicircle vectors with improved expression |
| AU2014363988B2 (en) | 2013-12-09 | 2020-06-11 | Baylor College Of Medicine | Hippo and dystrophin complex signaling in cardiomyocyte renewal |
| EP3097197B1 (en) | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| US10570183B2 (en) * | 2016-04-19 | 2020-02-25 | The Board Of Regents Of The University Of Texas System | Use of a small native peptide activator of SERCA pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload |
| JP2020515572A (ja) | 2017-03-27 | 2020-05-28 | ブレイエ・ユニバージテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ | 横隔膜特異的核酸調節エレメントならびにその方法および使用 |
| WO2020084162A1 (en) | 2018-10-26 | 2020-04-30 | Vrije Universiteit Brussel | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| EP4073257A1 (en) * | 2019-12-09 | 2022-10-19 | UCL Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4081643A1 (en) * | 2019-12-24 | 2022-11-02 | Asklepios Biopharmaceutical, Inc. | Regulatory nucleic acid sequences |
| EP4107259A1 (en) | 2020-02-18 | 2022-12-28 | Vrije Universiteit Brussel | Novel combination of nucleic acid regulatory elements and methods and uses thereof |
| EP4263828A1 (en) | 2020-12-21 | 2023-10-25 | Vrije Universiteit Brussel | Cardiomyocyte-derived nucleic acid regulatory elements and methods and use thereof |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2797273B1 (fr) * | 1999-08-06 | 2004-01-02 | Pasteur Institut | Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique |
| EP1250349A4 (en) * | 2000-01-14 | 2005-10-19 | Beth Israel Hospital | CARDIAC CELL SPECIFIC ACTIVATORY ELEMENTS AND USES THEREFOR |
| DE60130465T2 (de) * | 2000-07-14 | 2008-06-12 | The Regents Of The University Of Michigan, Ann Arbor | Mutierte muskelspezifische enhancer |
| US20050004058A1 (en) * | 2000-12-07 | 2005-01-06 | Patrick Benoit | Sequences upstream of the carp gene, vectors containing them and uses thereof |
| US7714100B2 (en) * | 2004-01-27 | 2010-05-11 | Compugen Ltd | Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of cardiac disease |
| CA2575614A1 (en) * | 2004-07-29 | 2006-02-09 | Stem Cell Innovations, Inc. | Differentiation of stem cells |
| US20080081354A1 (en) * | 2006-10-02 | 2008-04-03 | Cardiac Pacemakers, Inc. | Devices, vectors and methods for inducible ischemia cardioprotection |
| US8383601B2 (en) | 2006-10-30 | 2013-02-26 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| WO2008126083A2 (en) * | 2007-04-11 | 2008-10-23 | Technion Research & Development Foundation Ltd. | Methods for identification and selection of human embryonic stem cell derived cells |
| WO2009130208A1 (en) | 2008-04-22 | 2009-10-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| EP2494058B1 (en) | 2009-10-29 | 2017-04-05 | Vib Vzw | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
-
2010
- 2010-10-29 EP EP10771459.4A patent/EP2494058B1/en not_active Not-in-force
- 2010-10-29 AU AU2010311376A patent/AU2010311376B2/en not_active Ceased
- 2010-10-29 JP JP2012535849A patent/JP5823969B2/ja not_active Expired - Fee Related
- 2010-10-29 CA CA2778945A patent/CA2778945A1/en not_active Abandoned
- 2010-10-29 US US13/504,614 patent/US9353164B2/en not_active Expired - Fee Related
- 2010-10-29 WO PCT/EP2010/066474 patent/WO2011051450A1/en active Application Filing
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