JP2016533709A5 - - Google Patents
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- JP2016533709A5 JP2016533709A5 JP2016517431A JP2016517431A JP2016533709A5 JP 2016533709 A5 JP2016533709 A5 JP 2016533709A5 JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016533709 A5 JP2016533709 A5 JP 2016533709A5
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- acid residue
- seq
- capsid protein
- aav2 capsid
- 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
Links
- 108090000565 Capsid Proteins Proteins 0.000 claims 29
- 102100023321 Ceruloplasmin Human genes 0.000 claims 29
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 26
- 229910052757 nitrogen Inorganic materials 0.000 claims 12
- 229910052717 sulfur Inorganic materials 0.000 claims 11
- 229910052739 hydrogen Inorganic materials 0.000 claims 10
- 239000013607 AAV vector Substances 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 9
- 229910052698 phosphorus Inorganic materials 0.000 claims 8
- 229910052700 potassium Inorganic materials 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 238000006467 substitution reaction Methods 0.000 claims 6
- 210000002845 virion Anatomy 0.000 claims 6
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 4
- 239000004473 Threonine Substances 0.000 claims 4
- 235000001014 amino acid Nutrition 0.000 claims 3
- 125000000539 amino acid group Chemical group 0.000 claims 3
- 150000001413 amino acids Chemical group 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 239000003112 inhibitor Substances 0.000 claims 3
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 230000026683 transduction Effects 0.000 claims 3
- 238000010361 transduction Methods 0.000 claims 3
- 229910052722 tritium Inorganic materials 0.000 claims 3
- 239000013598 vector Substances 0.000 claims 3
- 229910052727 yttrium Inorganic materials 0.000 claims 3
- 239000004475 Arginine Substances 0.000 claims 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 2
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Natural products NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 2
- 239000004471 Glycine Substances 0.000 claims 2
- 239000004472 Lysine Substances 0.000 claims 2
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 2
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims 2
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 2
- 102000008847 Serpin Human genes 0.000 claims 2
- 108050000761 Serpin Proteins 0.000 claims 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 2
- 235000004279 alanine Nutrition 0.000 claims 2
- -1 antibody Proteins 0.000 claims 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 2
- 229960001230 asparagine Drugs 0.000 claims 2
- 235000009582 asparagine Nutrition 0.000 claims 2
- 235000013922 glutamic acid Nutrition 0.000 claims 2
- 239000004220 glutamic acid Substances 0.000 claims 2
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 2
- 239000003102 growth factor Substances 0.000 claims 2
- 229960000310 isoleucine Drugs 0.000 claims 2
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Natural products CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 claims 2
- 102000005962 receptors Human genes 0.000 claims 2
- 108020003175 receptors Proteins 0.000 claims 2
- 239000003001 serine protease inhibitor Substances 0.000 claims 2
- 125000000341 threoninyl group Chemical group [H]OC([H])(C([H])([H])[H])C([H])(N([H])[H])C(*)=O 0.000 claims 2
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 2
- 239000004474 valine Substances 0.000 claims 2
- 102000040125 5-hydroxytryptamine receptor family Human genes 0.000 claims 1
- 108091032151 5-hydroxytryptamine receptor family Proteins 0.000 claims 1
- 102000004031 Carboxy-Lyases Human genes 0.000 claims 1
- 108090000489 Carboxy-Lyases Proteins 0.000 claims 1
- 102000014914 Carrier Proteins Human genes 0.000 claims 1
- 108010078791 Carrier Proteins Proteins 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 101710112752 Cytotoxin Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
- 102000005755 Intercellular Signaling Peptides and Proteins Human genes 0.000 claims 1
- 108010070716 Intercellular Signaling Peptides and Proteins Proteins 0.000 claims 1
- 102000001617 Interferon Receptors Human genes 0.000 claims 1
- 102000015696 Interleukins Human genes 0.000 claims 1
- 108010063738 Interleukins Proteins 0.000 claims 1
- 241001529936 Murinae Species 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 1
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108091005804 Peptidases Proteins 0.000 claims 1
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 239000004365 Protease Substances 0.000 claims 1
- 229940124158 Protease/peptidase inhibitor Drugs 0.000 claims 1
- 102000001253 Protein Kinase Human genes 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 101710146873 Receptor-binding protein Proteins 0.000 claims 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 239000004393 Stigmasterol-rich plant sterol Substances 0.000 claims 1
- 102000001742 Tumor Suppressor Proteins Human genes 0.000 claims 1
- 108010040002 Tumor Suppressor Proteins Proteins 0.000 claims 1
- 239000000556 agonist Substances 0.000 claims 1
- 239000005557 antagonist Substances 0.000 claims 1
- 230000002424 anti-apoptotic effect Effects 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 239000002246 antineoplastic agent Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 229940009098 aspartate Drugs 0.000 claims 1
- 235000003704 aspartic acid Nutrition 0.000 claims 1
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 1
- 210000000601 blood cell Anatomy 0.000 claims 1
- 210000002798 bone marrow cell Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 102000003675 cytokine receptors Human genes 0.000 claims 1
- 108010057085 cytokine receptors Proteins 0.000 claims 1
- 229940127089 cytotoxic agent Drugs 0.000 claims 1
- 231100000599 cytotoxic agent Toxicity 0.000 claims 1
- 239000002619 cytotoxin Substances 0.000 claims 1
- 210000004443 dendritic cell Anatomy 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 210000002889 endothelial cell Anatomy 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 210000002919 epithelial cell Anatomy 0.000 claims 1
- 230000000913 erythropoietic effect Effects 0.000 claims 1
- 210000002950 fibroblast Anatomy 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 210000002064 heart cell Anatomy 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 108091008039 hormone receptors Proteins 0.000 claims 1
- 102000002467 interleukin receptors Human genes 0.000 claims 1
- 108010093036 interleukin receptors Proteins 0.000 claims 1
- 230000000968 intestinal effect Effects 0.000 claims 1
- 210000003292 kidney cell Anatomy 0.000 claims 1
- 229940043355 kinase inhibitor Drugs 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000005229 liver cell Anatomy 0.000 claims 1
- 210000005265 lung cell Anatomy 0.000 claims 1
- 239000000395 magnesium oxide Substances 0.000 claims 1
- 229940053128 nerve growth factor Drugs 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 claims 1
- 108010011903 peptide receptors Proteins 0.000 claims 1
- 102000014187 peptide receptors Human genes 0.000 claims 1
- COLNVLDHVKWLRT-QMMMGPOBSA-N phenylalanine group Chemical group N[C@@H](CC1=CC=CC=C1)C(=O)O COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 239000000441 potassium aluminium silicate Substances 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108060006633 protein kinase Proteins 0.000 claims 1
- 239000003909 protein kinase inhibitor Substances 0.000 claims 1
- 235000018102 proteins Nutrition 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 210000005167 vascular cell Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361883063P | 2013-09-26 | 2013-09-26 | |
| US61/883,063 | 2013-09-26 | ||
| PCT/US2014/057842 WO2015048534A1 (en) | 2013-09-26 | 2014-09-26 | Synthetic combinatorial aav capsid library for targeted gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019137247A Division JP7007337B2 (ja) | 2013-09-26 | 2019-07-25 | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533709A JP2016533709A (ja) | 2016-11-04 |
| JP2016533709A5 true JP2016533709A5 (cg-RX-API-DMAC7.html) | 2017-09-21 |
| JP6985795B2 JP6985795B2 (ja) | 2021-12-22 |
Family
ID=52744537
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016517431A Active JP6985795B2 (ja) | 2013-09-26 | 2014-09-26 | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
| JP2019137247A Active JP7007337B2 (ja) | 2013-09-26 | 2019-07-25 | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019137247A Active JP7007337B2 (ja) | 2013-09-26 | 2019-07-25 | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US20160369298A1 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3049527A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6985795B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2014324717B2 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2015048534A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (81)
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| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| JP6072772B2 (ja) | 2011-04-22 | 2017-02-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 |
| TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ） |
| WO2014194132A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
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2014
- 2014-09-26 AU AU2014324717A patent/AU2014324717B2/en active Active
- 2014-09-26 EP EP14848603.8A patent/EP3049527A4/en not_active Ceased
- 2014-09-26 US US15/024,431 patent/US20160369298A1/en not_active Abandoned
- 2014-09-26 JP JP2016517431A patent/JP6985795B2/ja active Active
- 2014-09-26 EP EP19207549.7A patent/EP3633041A3/en active Pending
- 2014-09-26 WO PCT/US2014/057842 patent/WO2015048534A1/en not_active Ceased
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2018
- 2018-12-03 US US16/208,127 patent/US11091777B2/en active Active
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2019
- 2019-07-25 JP JP2019137247A patent/JP7007337B2/ja active Active
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