JP2020010684A5 - - Google Patents
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- JP2020010684A5 JP2020010684A5 JP2019135745A JP2019135745A JP2020010684A5 JP 2020010684 A5 JP2020010684 A5 JP 2020010684A5 JP 2019135745 A JP2019135745 A JP 2019135745A JP 2019135745 A JP2019135745 A JP 2019135745A JP 2020010684 A5 JP2020010684 A5 JP 2020010684A5
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- capsid protein
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- 125000003275 alpha amino acid group Chemical group 0.000 claims 30
- 210000002845 Virion Anatomy 0.000 claims 17
- 102000004040 Capsid Proteins Human genes 0.000 claims 16
- 108090000565 Capsid Proteins Proteins 0.000 claims 16
- 230000002458 infectious Effects 0.000 claims 14
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 150000007523 nucleic acids Chemical class 0.000 claims 12
- 210000004027 cells Anatomy 0.000 claims 11
- 150000001413 amino acids Chemical class 0.000 claims 9
- 210000000130 stem cell Anatomy 0.000 claims 9
- 241000432074 Adeno-associated virus Species 0.000 claims 8
- 108090001123 antibodies Proteins 0.000 claims 8
- 102000004965 antibodies Human genes 0.000 claims 8
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 4
- 230000005591 charge neutralization Effects 0.000 claims 4
- 210000004962 mammalian cells Anatomy 0.000 claims 4
- 230000001264 neutralization Effects 0.000 claims 4
- 238000006386 neutralization reaction Methods 0.000 claims 4
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 230000026683 transduction Effects 0.000 claims 3
- 238000010361 transduction Methods 0.000 claims 3
- 210000003958 Hematopoietic Stem Cells Anatomy 0.000 claims 2
- 210000004185 Liver Anatomy 0.000 claims 2
- 108010061543 Neutralizing Antibodies Proteins 0.000 claims 2
- 229930002945 all-trans-retinaldehyde Natural products 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 230000002207 retinal Effects 0.000 claims 2
- 235000020945 retinal Nutrition 0.000 claims 2
- 239000011604 retinal Substances 0.000 claims 2
- 210000001671 Embryonic Stem Cells Anatomy 0.000 claims 1
- 210000002889 Endothelial Cells Anatomy 0.000 claims 1
- 210000004263 Induced Pluripotent Stem Cells Anatomy 0.000 claims 1
- 210000004072 Lung Anatomy 0.000 claims 1
- 210000002901 Mesenchymal Stem Cells Anatomy 0.000 claims 1
- 210000004413 Myocytes, Cardiac Anatomy 0.000 claims 1
- 210000000933 Neural Crest Anatomy 0.000 claims 1
- 210000001178 Neural Stem Cells Anatomy 0.000 claims 1
- 210000000496 Pancreas Anatomy 0.000 claims 1
- 210000001744 T-Lymphocytes Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 210000002317 cardiac myocyte Anatomy 0.000 claims 1
- 201000009910 diseases by infectious agent Diseases 0.000 claims 1
- 210000002950 fibroblast Anatomy 0.000 claims 1
- 210000001665 muscle stem cell Anatomy 0.000 claims 1
- 230000001537 neural Effects 0.000 claims 1
- 210000004255 neuroglia Anatomy 0.000 claims 1
- 210000002569 neurons Anatomy 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001568 sexual Effects 0.000 claims 1
- 210000002363 skeletal muscle cell Anatomy 0.000 claims 1
Claims (29)
- (a)配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも90%のアミノ酸配列同一性を有するアミノ酸配列を含む、変異体アデノ随伴ウイルス(AAV)カプシドタンパク質、ならびに
(b)異種核酸
を含み、AAV2によって示されるヒト中和抗体への耐性と比較して、少なくとも5倍高い該抗体への耐性を示す、感染性組換えアデノ随伴ウイルス(rAAV)ビリオン。 - 前記変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも95%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項1に記載の前記感染性rAAVビリオン。
- 前記変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも98%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項1に記載の前記感染性rAAVビリオン。
- 前記変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列を含む、請求項1に記載の前記感染性rAAVビリオン。
- 前記rAAVビリオンが、AAV2によって示される耐性よりも少なくとも10倍高いヒト中和抗体への耐性を示す、請求項1〜4のいずれか一項に記載の前記感染性rAAVビリオン。
- 前記rAAVビリオンが、AAV2によって示される耐性よりも少なくとも30倍高いヒト中和抗体への耐性を示す、請求項1〜4のいずれか一項に記載の前記感染性rAAVビリオン。
- 野生型AAV血清型2(AAV2)によって示される哺乳類細胞への形質導入と比較して、ヒト中和抗体の存在下における哺乳類細胞への増加した形質導入を示す、請求項1〜6のいずれか一項に記載の前記感染性rAAVビリオン。
- 前記哺乳類細胞が、肝臓細胞、膵臓細胞、骨格筋細胞、心筋細胞、線維芽細胞、網膜細胞、滑膜関節細胞、肺細胞、T細胞、ニューロン、グリア細胞、幹細胞、内皮細胞、または癌細胞である、請求項7に記載の前記感染性rAAVビリオン。
- 前記幹細胞が、造血幹細胞、造血前駆細胞、神経幹細胞、神経前駆細胞、神経堤幹細胞、胚幹細胞、誘導多能性幹細胞(iPS細胞)、間葉幹細胞、中胚葉幹細胞、肝臓幹細胞、膵臓幹細胞、膵臓前駆細胞、筋幹細胞、または網膜幹細胞である、請求項8に記載の前記感染性rAAVビリオン。
- 前記異種核酸が干渉RNAを含む、請求項1〜9のいずれか一項に記載の前記感染性rAAVビリオン。
- 前記異種核酸が、ポリペプチドをコードするヌクレオチド配列を含む、請求項1〜9のいずれか一項に記載の前記感染性rAAVビリオン。
- 配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも90%のアミノ酸配列同一性を有するアミノ酸配列を含む変異体アデノ随伴ウイルス(AAV)カプシドタンパク質をコードするヌクレオチド配列を含み、該コードされた変異体AAVカプシドタンパク質が、AAV2によって示されるヒト抗体による中和への耐性と比較して、少なくとも5倍上昇した、該抗体による中和への耐性を、感染性組換えアデノ随伴ウイルス(rAAV)ビリオンに与える、単離核酸。
- 前記コードされた変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも95%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項12に記載の前記単離核酸。
- 前記コードされた変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも98%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項12に記載の前記単離核酸。
- 前記コードされた変異体AAVカプシドタンパク質が、配列番号11または配列番号12に記載されるアミノ酸配列を含む、請求項12に記載の前記単離核酸。
- 請求項12〜15のいずれか一項に記載の前記核酸を含む、単離宿主細胞。
- 前記核酸で安定的にトランスフェクトされる、請求項16に記載の前記単離宿主細胞。
- AAVrepタンパク質をコードするヌクレオチド配列を含む核酸をさらに含む、請求項16または17に記載の前記単離宿主細胞。
- 組換えAAVベクターをさらに含む、請求項16〜18のいずれか一項に記載の前記単離宿主細胞。
- 請求項1〜11のいずれか一項に記載の感染性組換えアデノ随伴ウイルス(rAAV)ビリオンを含む、遺伝子産物の送達剤。
- 前記異種核酸が干渉RNAを含む、請求項20に記載の前記送達剤。
- 前記異種核酸がポリペプチドをコードするヌクレオチド配列を含む、請求項20に記載の前記送達剤。
- 配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも90%のアミノ酸配列同一性を有するアミノ酸配列を含み、AAV2によって示されるヒト抗体による中和への耐性と比較して、少なくとも5倍上昇した、該抗体による中和への耐性を、感染性組換えアデノ随伴ウイルスビリオンに与える、変異体アデノ随伴ウイルス(AAV)カプシドタンパク質。
- 配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも95%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項23に記載の前記変異体AAVカプシドタンパク質。
- 配列番号11または配列番号12に記載されるアミノ酸配列のアミノ酸203〜736と少なくとも98%のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項23に記載の前記変異体AAVカプシドタンパク質。
- 配列番号11または配列番号12に記載されるアミノ酸配列を含む、請求項23に記載の前記変異体AAVカプシドタンパク質。
- 前記上昇した耐性が、AAV2によって示される耐性よりも少なくとも10倍高い、請求項23〜26のいずれか一項に記載の前記変異体AAVカプシドタンパク質。
- 前記上昇した耐性が、AAV2によって示される耐性よりも少なくとも30倍高い、請求項23〜26のいずれか一項に記載の前記変異体AAVカプシドタンパク質。
- AAV2によって示される形質導入と比較して、ヒト中和抗体の存在下における哺乳類細胞への増加した形質導入を、感染性組換えアデノ随伴ウイルス(rAAV)ビリオンに与える、請求項23〜28のいずれか一項に記載の前記変異体AAVカプシドタンパク質。
Priority Applications (2)
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JP2022052809A JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
JP2024006465A JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
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US201361829735P | 2013-05-31 | 2013-05-31 | |
US61/829,735 | 2013-05-31 |
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JP2016517017A Division JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
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JP2022052809A Division JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
Publications (3)
Publication Number | Publication Date |
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JP2020010684A JP2020010684A (ja) | 2020-01-23 |
JP2020010684A5 true JP2020010684A5 (ja) | 2020-05-21 |
JP7069088B2 JP7069088B2 (ja) | 2022-05-17 |
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JP2016517017A Active JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
JP2019135745A Active JP7069088B2 (ja) | 2013-05-31 | 2019-07-24 | アデノ随伴ウイルス変異体及びその使用方法 |
JP2022052809A Pending JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
JP2024006465A Pending JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
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JP2016517017A Active JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
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JP2022052809A Pending JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
JP2024006465A Pending JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
Country Status (8)
Country | Link |
---|---|
US (4) | US11136557B2 (ja) |
EP (1) | EP3003391B1 (ja) |
JP (4) | JP6600624B2 (ja) |
CN (1) | CN105247044B (ja) |
CA (1) | CA2907799A1 (ja) |
ES (1) | ES2897508T3 (ja) |
HK (1) | HK1223016A1 (ja) |
WO (1) | WO2014194132A1 (ja) |
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