JP2008545406A5 - - Google Patents
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- JP2008545406A5 JP2008545406A5 JP2008512952A JP2008512952A JP2008545406A5 JP 2008545406 A5 JP2008545406 A5 JP 2008545406A5 JP 2008512952 A JP2008512952 A JP 2008512952A JP 2008512952 A JP2008512952 A JP 2008512952A JP 2008545406 A5 JP2008545406 A5 JP 2008545406A5
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- 108020004388 MicroRNAs Proteins 0.000 claims 22
- 239000002679 microRNA Substances 0.000 claims 22
- 229920001239 microRNA Polymers 0.000 claims 22
- 239000002245 particle Substances 0.000 claims 17
- 230000003612 virological Effects 0.000 claims 15
- 210000004027 cells Anatomy 0.000 claims 11
- 239000008194 pharmaceutical composition Substances 0.000 claims 10
- 239000003814 drug Substances 0.000 claims 7
- 238000004519 manufacturing process Methods 0.000 claims 7
- 230000000875 corresponding Effects 0.000 claims 4
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims 4
- 230000002265 prevention Effects 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 230000001225 therapeutic Effects 0.000 claims 3
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 201000011510 cancer Diseases 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 230000001404 mediated Effects 0.000 claims 2
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims 1
- 241000432074 Adeno-associated virus Species 0.000 claims 1
- 241000710929 Alphavirus Species 0.000 claims 1
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 206010003246 Arthritis Diseases 0.000 claims 1
- 210000001671 Embryonic Stem Cells Anatomy 0.000 claims 1
- 206010061992 Haemophilia Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 208000009889 Herpes Simplex Diseases 0.000 claims 1
- 229940088597 Hormone Drugs 0.000 claims 1
- 206010021425 Immune system disease Diseases 0.000 claims 1
- 210000004185 Liver Anatomy 0.000 claims 1
- 210000004072 Lung Anatomy 0.000 claims 1
- 229920001922 Mir-145 Polymers 0.000 claims 1
- 241000709664 Picornaviridae Species 0.000 claims 1
- 241000700584 Simplexvirus Species 0.000 claims 1
- 210000000952 Spleen Anatomy 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 102000001742 Tumor Suppressor Proteins Human genes 0.000 claims 1
- 108010040002 Tumor Suppressor Proteins Proteins 0.000 claims 1
- 206010047461 Viral infection Diseases 0.000 claims 1
- 208000001756 Virus Disease Diseases 0.000 claims 1
- 230000003213 activating Effects 0.000 claims 1
- 230000000840 anti-viral Effects 0.000 claims 1
- 102000004965 antibodies Human genes 0.000 claims 1
- 108090001123 antibodies Proteins 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000038129 antigens Human genes 0.000 claims 1
- 108091007172 antigens Proteins 0.000 claims 1
- 229920002847 antisense RNA Polymers 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 102000037240 fusion proteins Human genes 0.000 claims 1
- 108020001507 fusion proteins Proteins 0.000 claims 1
- 201000002406 genetic disease Diseases 0.000 claims 1
- 238000010353 genetic engineering Methods 0.000 claims 1
- 230000003394 haemopoietic Effects 0.000 claims 1
- 210000002064 heart cell Anatomy 0.000 claims 1
- 201000010238 heart disease Diseases 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 230000002519 immonomodulatory Effects 0.000 claims 1
- 230000028993 immune response Effects 0.000 claims 1
- 210000003292 kidney cell Anatomy 0.000 claims 1
- 239000012528 membrane Substances 0.000 claims 1
- 230000000051 modifying Effects 0.000 claims 1
- 210000003867 nerve cell Anatomy 0.000 claims 1
- 230000000926 neurological Effects 0.000 claims 1
- 210000002569 neurons Anatomy 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 239000000651 prodrug Substances 0.000 claims 1
- 229940002612 prodrugs Drugs 0.000 claims 1
- 229920002033 ribozyme Polymers 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 210000001519 tissues Anatomy 0.000 claims 1
- 210000004881 tumor cells Anatomy 0.000 claims 1
- 239000000225 tumor suppressor protein Substances 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
- 230000002227 vasoactive Effects 0.000 claims 1
- 230000017613 viral reproduction Effects 0.000 claims 1
Claims (33)
- miRNA配列標的および必要に応じてトランスジーンを含む遺伝子操作における使用のための遺伝子ベクター。
- 治療における使用のためのmiRNA配列標的を含む遺伝子ベクター。
- 非ウイルス遺伝子ベクターの形態である、請求項１または２に記載の遺伝子ベクター。
- 非ウイルス遺伝子ベクターが、miRNA配列標的および必要に応じて少なくとも1個のトランスジーンを含む発現ベクターまたはプラスミドを含む、請求項３に記載の遺伝子ベクター。
- miRNA配列標的を含むウイルスベクターの形態である、請求項１または２に記載の遺伝子ベクター。
- ゲノムがmiRNA配列標的を含む、前記ウイルスベクターのゲノム(RNAまたはDNA)を含む請求項５に記載の遺伝子ベクター。
- ゲノムが、miRNA配列標的に機能し得る形で連結された少なくとも1個のトランスジーンを含む、ウイルスベクターのゲノムを含む請求項６に記載の遺伝子ベクター。
- ウイルスベクターが、レトロウイルス、レンチウイルス、アデノウイルス、アデノ随伴ウイルス、単純ヘルペスウイルス、ピコルナウイルス、およびアルファウイルスの群から選択される、請求項５〜７のいずれか１項に記載の遺伝子ベクター。
- ウイルスベクターがレンチウイルスから誘導可能である、請求項５〜７のいずれか１項に記載の遺伝子ベクター。
- ウイルスベクターがウイルスベクター粒子の形態である、請求項５〜８のいずれか１項に記載の遺伝子ベクター。
- miRNA配列標的が、標的細胞中のウイルスベクターの発現を制御する、請求項１〜１０のいずれか１項に記載の遺伝子ベクター。
- 必要に応じて直列配向および/または必要に応じて異なる配向の、同じであっても異なっていてもよい2つ以上のmiRNA配列標的を含む、請求項１〜１１のいずれか１項に記載の遺伝子ベクター。
- miRNA配列標的が、has-mir-142as(has-mir-142-3pとも呼ばれる) miRNA、let-7a、mir-15a、mir-16、mir-17-5p、mir-19、mir-142-5p、mir-145および／またはmir-218 miRNAにより標的化されるものである、請求項１〜１２のいずれか１項に記載の遺伝子ベクター。
- 好適な治療遺伝子をコードするトランスジーンが、腫瘍抑制タンパク質、酵素、プロドラッグ活性化酵素、免疫調節分子、抗体、操作された免疫グロブリン様分子、融合タンパク質、ホルモン、膜タンパク質、血管作用タンパク質もしくはペプチド、サイトカイン、ケモカイン、抗ウイルスタンパク質、アンチセンスRNAおよびリボザイムをコードするトランスジーンを含む、請求項１〜１３のいずれか１項に記載の遺伝子ベクター。
- ベクターが組織特異的プロモーターを含む、請求項１〜１４のいずれか１項に記載の遺伝子ベクター。
- miRNA配列標的、および必要に応じてトランスジーンを含むパッケージング可能なウイルスベクターゲノムをコードするDNA構築物を含むウイルスベクター粒子を製造するためのDNA構築物のセット。
- 請求項１６に記載のDNA構築物のセットを宿主細胞に導入し、ウイルスベクター粒子を取得することを含む、ウイルスベクター粒子を調製する方法。
- 宿主細胞が対応するmiRNAを含む、請求項１７に記載の方法。
- 請求項１７または１８に記載の方法により製造されたウイルスベクター粒子。
- 請求項１〜１５または１９のいずれか１項に記載の遺伝子ベクターまたは粒子を含む医薬組成物。
- 請求項１〜１５または１９のいずれか１項に記載の遺伝子ベクターまたは粒子を用いて感染または形質導入された細胞。
- 造血細胞、腎臓細胞、神経細胞、肺細胞、肝臓細胞、脾臓細胞、心臓細胞、腫瘍細胞または胚性幹細胞である、請求項２１に記載の細胞。
- 対応するmiRNAを含む細胞中のトランスジーンの発現を防止するか、または減少させるための、miRNA標的配列に機能し得る形で連結されたトランスジーンを含む、請求項１〜１５のいずれか１項に記載のベクターの、請求項１９に記載の粒子のまたは請求項２０に記載の医薬組成物の、使用。
- 遺伝子治療における使用のために対応するmiRNAを含む細胞中のトランスジーンの発現を防止するか、または減少させるための、miRNA標的配列に機能し得る形で連結されたトランスジーンを含む、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物。
- miRNA標的配列に機能し得る形で連結されたトランスジーンを含む、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の、癌治療用の医薬の製造のための使用。
- miRNA標的配列に機能し得る形で連結されたトランスジーンを含む、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の、血友病治療用の医薬の製造のための使用。
- miRNA標的配列に機能し得る形で連結されたトランスジーンを含む、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の、導入された遺伝子の免疫介在性拒絶の予防用の医薬の製造のための使用。
- 導入された遺伝子の免疫介在性拒絶の予防用の医薬の製造のための、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の使用。
- 循環抗原に対する免疫応答の予防用の医薬の製造のための、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の使用。
- 癌、神経疾患、遺伝病、心疾患、卒中、関節炎、ウイルス感染および免疫系の疾患から選択される疾患の治療用の医薬の製造のための、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の使用。
- 遺伝子治療用の医薬の製造のための、請求項１〜１５のいずれか１項に記載の遺伝子ベクター、請求項１９に記載の粒子または請求項２０に記載の医薬組成物の使用。
- 治療用途に使用するための、miRNA標的配列の使用。
- 治療用途が、対応する内因性miRNAを用いて遺伝子を調節することを含む、請求項３２に記載の使用。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US68495405P | 2005-05-27 | 2005-05-27 | |
| US60/684,954 | 2005-05-27 | ||
| PCT/IB2006/002266 WO2007000668A2 (en) | 2005-05-27 | 2006-05-26 | Gene vector comprising mi-rna |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013098229A Division JP2013226139A (ja) | 2005-05-27 | 2013-05-08 | 遺伝子ベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2008545406A JP2008545406A (ja) | 2008-12-18 |
| JP2008545406A5 true JP2008545406A5 (ja) | 2009-07-09 |
| JP5956709B2 JP5956709B2 (ja) | 2016-07-27 |
Family
ID=37595503
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008512952A Active JP5956709B2 (ja) | 2005-05-27 | 2006-05-26 | 遺伝子ベクター |
| JP2013098229A Withdrawn JP2013226139A (ja) | 2005-05-27 | 2013-05-08 | 遺伝子ベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013098229A Withdrawn JP2013226139A (ja) | 2005-05-27 | 2013-05-08 | 遺伝子ベクター |
Country Status (14)
| Country | Link |
|---|---|
| US (4) | US10000757B2 (ja) |
| EP (2) | EP3002330A1 (ja) |
| JP (2) | JP5956709B2 (ja) |
| KR (2) | KR20130114758A (ja) |
| CN (1) | CN101287834B (ja) |
| CA (1) | CA2609142C (ja) |
| DK (1) | DK2002003T3 (ja) |
| ES (1) | ES2564823T3 (ja) |
| HU (1) | HUE027013T2 (ja) |
| IL (1) | IL187679A (ja) |
| PL (1) | PL2002003T3 (ja) |
| SG (1) | SG162726A1 (ja) |
| SI (1) | SI2002003T1 (ja) |
| WO (1) | WO2007000668A2 (ja) |
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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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| EP2125032A4 (en) | 2007-02-20 | 2011-02-23 | Mayo Foundation | TREATMENT OF CANCER USING VIRAL NUCLEIC ACID |
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