JP2016533709A5 - - Google Patents
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- Publication number
- 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 (enExample) | 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 (6)
| Country | Link |
|---|---|
| US (2) | US20160369298A1 (enExample) |
| EP (2) | EP3049527A4 (enExample) |
| JP (2) | JP6985795B2 (enExample) |
| AU (1) | AU2014324717B2 (enExample) |
| CA (1) | CA2930549A1 (enExample) |
| WO (1) | WO2015048534A1 (enExample) |
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 |
| MX349138B (es) | 2011-04-22 | 2017-07-13 | Univ California | Variones de virus adeno-asociados con capside variante y sus metodos de uso. |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 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) |
| CA2907799A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| EP3049527A4 (en) | 2013-09-26 | 2017-08-16 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| KR102288849B1 (ko) | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
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2014
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- 2014-09-26 CA CA2930549A patent/CA2930549A1/en active Pending
- 2014-09-26 JP JP2016517431A patent/JP6985795B2/ja active Active
- 2014-09-26 EP EP19207549.7A patent/EP3633041A3/en active Pending
- 2014-09-26 AU AU2014324717A patent/AU2014324717B2/en active Active
- 2014-09-26 US US15/024,431 patent/US20160369298A1/en not_active Abandoned
- 2014-09-26 WO PCT/US2014/057842 patent/WO2015048534A1/en not_active Ceased
-
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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