JP2020510418A5 - - Google Patents
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- JP2020510418A5 JP2020510418A5 JP2019545322A JP2019545322A JP2020510418A5 JP 2020510418 A5 JP2020510418 A5 JP 2020510418A5 JP 2019545322 A JP2019545322 A JP 2019545322A JP 2019545322 A JP2019545322 A JP 2019545322A JP 2020510418 A5 JP2020510418 A5 JP 2020510418A5
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- JP
- Japan
- Prior art keywords
- seq
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- animal
- polypeptide
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- 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.)
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- 108010033576 Transferrin Receptors Proteins 0.000 claims description 70
- 102000007238 Transferrin Receptors Human genes 0.000 claims description 65
- 238000000034 method Methods 0.000 claims description 57
- 229920001184 polypeptide Polymers 0.000 claims description 54
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 54
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 54
- 241001465754 Metazoa Species 0.000 claims description 50
- 210000004027 cell Anatomy 0.000 claims description 39
- 230000009261 transgenic effect Effects 0.000 claims description 34
- 239000012636 effector Substances 0.000 claims description 24
- 108091033319 polynucleotide Proteins 0.000 claims description 24
- 102000040430 polynucleotide Human genes 0.000 claims description 24
- 239000002157 polynucleotide Substances 0.000 claims description 24
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 22
- 210000001161 mammalian embryo Anatomy 0.000 claims description 16
- 210000004556 brain Anatomy 0.000 claims description 15
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 11
- 150000007523 nucleic acids Chemical group 0.000 claims description 11
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 9
- 239000000427 antigen Substances 0.000 claims description 9
- 102000036639 antigens Human genes 0.000 claims description 9
- 108091007433 antigens Proteins 0.000 claims description 9
- 239000012634 fragment Substances 0.000 claims description 9
- 239000002773 nucleotide Substances 0.000 claims description 9
- 125000003729 nucleotide group Chemical group 0.000 claims description 9
- 108020004414 DNA Proteins 0.000 claims description 8
- 230000000694 effects Effects 0.000 claims description 6
- 102000004338 Transferrin Human genes 0.000 claims description 5
- 108090000901 Transferrin Proteins 0.000 claims description 5
- 230000003285 pharmacodynamic effect Effects 0.000 claims description 5
- 239000012581 transferrin Substances 0.000 claims description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 230000008499 blood brain barrier function Effects 0.000 claims description 4
- 210000001218 blood-brain barrier Anatomy 0.000 claims description 4
- 150000003384 small molecules Chemical class 0.000 claims description 4
- 241000283984 Rodentia Species 0.000 claims description 3
- 238000012216 screening Methods 0.000 claims description 3
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 108091026890 Coding region Proteins 0.000 claims description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 2
- 210000005013 brain tissue Anatomy 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 229940079593 drug Drugs 0.000 claims description 2
- 210000001671 embryonic stem cell Anatomy 0.000 claims description 2
- 230000006801 homologous recombination Effects 0.000 claims description 2
- 238000002744 homologous recombination Methods 0.000 claims description 2
- 238000012544 monitoring process Methods 0.000 claims description 2
- 101000835089 Mus musculus Transferrin receptor protein 1 Proteins 0.000 claims 5
- 102000001554 Hemoglobins Human genes 0.000 claims 2
- 108010054147 Hemoglobins Proteins 0.000 claims 2
- 210000002257 embryonic structure Anatomy 0.000 claims 2
- 238000005534 hematocrit Methods 0.000 claims 2
- 241000894007 species Species 0.000 claims 2
- 238000006467 substitution reaction Methods 0.000 claims 2
- 238000004820 blood count Methods 0.000 claims 1
- 210000003743 erythrocyte Anatomy 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000005259 measurement Methods 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 101100369221 Mus musculus Tfrc gene Proteins 0.000 description 5
- 238000003018 immunoassay Methods 0.000 description 4
- 210000003038 endothelium Anatomy 0.000 description 3
- 238000002198 surface plasmon resonance spectroscopy Methods 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 1
- 101000766306 Homo sapiens Serotransferrin Proteins 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- 101000766305 Mus musculus Serotransferrin Proteins 0.000 description 1
- 239000003795 chemical substances by application Substances 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- XNSAINXGIQZQOO-SRVKXCTJSA-N protirelin Chemical compound NC(=O)[C@@H]1CCCN1C(=O)[C@@H](NC(=O)[C@H]1NC(=O)CC1)CC1=CN=CN1 XNSAINXGIQZQOO-SRVKXCTJSA-N 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023085382A JP2023116513A (ja) | 2017-02-17 | 2023-05-24 | トランスフェリン受容体トランスジェニックモデル |
| JP2025036281A JP2025087857A (ja) | 2017-02-17 | 2025-03-07 | トランスフェリン受容体トランスジェニックモデル |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762460692P | 2017-02-17 | 2017-02-17 | |
| US62/460,692 | 2017-02-17 | ||
| US201762543559P | 2017-08-10 | 2017-08-10 | |
| US201762543658P | 2017-08-10 | 2017-08-10 | |
| US62/543,559 | 2017-08-10 | ||
| US62/543,658 | 2017-08-10 | ||
| US201762583314P | 2017-11-08 | 2017-11-08 | |
| US62/583,314 | 2017-11-08 | ||
| PCT/US2018/018302 WO2018152285A1 (en) | 2017-02-17 | 2018-02-15 | Transferrin receptor transgenic models |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023085382A Division JP2023116513A (ja) | 2017-02-17 | 2023-05-24 | トランスフェリン受容体トランスジェニックモデル |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020510418A JP2020510418A (ja) | 2020-04-09 |
| JP2020510418A5 true JP2020510418A5 (enExample) | 2021-03-11 |
| JP7669113B2 JP7669113B2 (ja) | 2025-04-28 |
Family
ID=61628463
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019545322A Active JP7669113B2 (ja) | 2017-02-17 | 2018-02-15 | トランスフェリン受容体トランスジェニックモデル |
| JP2023085382A Pending JP2023116513A (ja) | 2017-02-17 | 2023-05-24 | トランスフェリン受容体トランスジェニックモデル |
| JP2025036281A Pending JP2025087857A (ja) | 2017-02-17 | 2025-03-07 | トランスフェリン受容体トランスジェニックモデル |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023085382A Pending JP2023116513A (ja) | 2017-02-17 | 2023-05-24 | トランスフェリン受容体トランスジェニックモデル |
| JP2025036281A Pending JP2025087857A (ja) | 2017-02-17 | 2025-03-07 | トランスフェリン受容体トランスジェニックモデル |
Country Status (9)
| Country | Link |
|---|---|
| EP (2) | EP4628586A2 (enExample) |
| JP (3) | JP7669113B2 (enExample) |
| KR (1) | KR102637590B1 (enExample) |
| CN (1) | CN110382526B (enExample) |
| AU (1) | AU2018221704B2 (enExample) |
| CA (1) | CA3053370A1 (enExample) |
| IL (1) | IL268659A (enExample) |
| SG (1) | SG11201907419PA (enExample) |
| WO (1) | WO2018152285A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| SG11202011743SA (en) | 2018-06-18 | 2021-01-28 | Denali Therapeutics Inc | Fusion proteins comprising progranulin |
| WO2021133907A1 (en) | 2019-12-23 | 2021-07-01 | Denali Therapeutics Inc. | Progranulin variants |
| CN112243953B (zh) * | 2020-07-15 | 2022-02-11 | 吉林医药学院 | TfR1肺组织特异性敲除小鼠模型及其构建方法和应用 |
| PE20231931A1 (es) | 2020-10-14 | 2023-12-01 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas |
| CN112501124A (zh) * | 2020-11-27 | 2021-03-16 | 领诺(上海)医药科技有限公司 | 稳定表达人转铁蛋白受体1细胞株的制备方法及其应用 |
| EP4370539A4 (en) * | 2021-07-16 | 2025-05-28 | Biocytogen Pharmaceuticals (Beijing) Co., Ltd. | Genetically modified non-human animal with human or chimeric tfr1 |
| US20250136963A1 (en) | 2021-12-17 | 2025-05-01 | Denali Therapeutics Inc. | Fusion proteins comprising alpha-l-iduronidase enzymes and methods |
| CA3242959A1 (en) | 2021-12-17 | 2025-02-26 | Denali Therapeutics Inc | POLYPEPTIDE ENGINEERING, LIBRARIES AND ENGINEERED POLYPEPTIDES FOR CD98 HEAVY CHAIN LINK AND TRANSFERRINE RECEIVER |
| US20250215077A1 (en) | 2022-03-28 | 2025-07-03 | Denali Therapeutics Inc. | Monovalent anti-trem2 binding molecules and methods of use thereof |
| KR20250048010A (ko) * | 2022-07-29 | 2025-04-07 | 리제너론 파마슈티칼스 인코포레이티드 | 변형된 트랜스페린 수용체 좌위를 포함하는 비-인간 동물 |
| EP4658684A1 (en) | 2023-01-30 | 2025-12-10 | ISAR Bioscience GmbH | Human anti-trem2 antibody for treating neurodegenerative disorders |
| CN116784280B (zh) * | 2023-08-10 | 2025-08-19 | 广东药康生物科技有限公司 | 一种tfrc人源化小鼠模型的构建方法及其应用 |
| WO2025137213A1 (en) | 2023-12-20 | 2025-06-26 | Denali Therapeutics Inc. | Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5898094A (en) | 1996-10-21 | 1999-04-27 | University Of South Florida | Transgenic mice expressing APPK670N,M671L and a mutant presenilin transgenes |
| US6175057B1 (en) | 1997-10-08 | 2001-01-16 | The Regents Of The University Of California | Transgenic mouse model of alzheimer's disease and cerebral amyloid angiopathy |
| EP1620564A4 (en) * | 2003-04-18 | 2008-03-12 | Cytovia Inc | METHODS FOR TREATING DISEASES INDUCING APOPTOSIS INDUCTION AND SCREENING ASSAYS |
| WO2009131632A1 (en) | 2008-04-14 | 2009-10-29 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| EP3028565B1 (en) | 2009-07-08 | 2017-09-27 | Kymab Limited | Animal models and therapeutic molecules |
| WO2011100058A1 (en) | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US8476485B2 (en) | 2010-06-24 | 2013-07-02 | Academia Sinica | Non-human animal model for amyotrophic lateral sclerosis (ALS) with loss-of-TDP-43 function |
| US9232775B2 (en) | 2012-08-03 | 2016-01-12 | Industry-Academic Cooperation Foundation, Yonsei University | Genetically engineered mouse model for autism spectrum disorder having deletion of Shank2 gene and use thereof |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| JP2016516399A (ja) | 2013-03-13 | 2016-06-09 | オーストラリアン ニュークリア サイエンス アンド テクノロジー オーガニゼーション | 非機能性tspo遺伝子を有するトランスジェニック非ヒト生物 |
| EP4324480A3 (en) * | 2013-05-20 | 2024-05-08 | F. Hoffmann-La Roche AG | Anti-transferrin receptor antibodies and methods of use |
| EP3988649B1 (en) | 2013-09-18 | 2024-11-27 | Kymab Limited | Methods, cells and organisms |
| CN103897033A (zh) * | 2014-03-31 | 2014-07-02 | 中国药科大学 | 转铁蛋白受体TfR特异性结合肽及其应用 |
| EP3221362B1 (en) * | 2014-11-19 | 2019-07-24 | F.Hoffmann-La Roche Ag | Anti-transferrin receptor antibodies and methods of use |
| CN107531788B (zh) * | 2015-06-24 | 2022-06-21 | 豪夫迈·罗氏有限公司 | 对her2和血脑屏障受体特异性的三特异性抗体及使用方法 |
| CA3053381A1 (en) * | 2017-02-17 | 2018-08-23 | Denali Therapeutics Inc. | Engineered polypeptides |
-
2018
- 2018-02-15 WO PCT/US2018/018302 patent/WO2018152285A1/en not_active Ceased
- 2018-02-15 CA CA3053370A patent/CA3053370A1/en active Pending
- 2018-02-15 CN CN201880012330.9A patent/CN110382526B/zh active Active
- 2018-02-15 AU AU2018221704A patent/AU2018221704B2/en active Active
- 2018-02-15 EP EP25180359.9A patent/EP4628586A2/en active Pending
- 2018-02-15 EP EP18711190.1A patent/EP3583118B1/en active Active
- 2018-02-15 JP JP2019545322A patent/JP7669113B2/ja active Active
- 2018-02-15 KR KR1020197025801A patent/KR102637590B1/ko active Active
- 2018-02-15 SG SG11201907419PA patent/SG11201907419PA/en unknown
-
2019
- 2019-08-12 IL IL26865919A patent/IL268659A/en unknown
-
2023
- 2023-05-24 JP JP2023085382A patent/JP2023116513A/ja active Pending
-
2025
- 2025-03-07 JP JP2025036281A patent/JP2025087857A/ja active Pending
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