JP2023071853A5 - - Google Patents
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- JP2023071853A5 JP2023071853A5 JP2023031534A JP2023031534A JP2023071853A5 JP 2023071853 A5 JP2023071853 A5 JP 2023071853A5 JP 2023031534 A JP2023031534 A JP 2023031534A JP 2023031534 A JP2023031534 A JP 2023031534A JP 2023071853 A5 JP2023071853 A5 JP 2023071853A5
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- Prior art keywords
- cells
- seq
- cell
- nanoparticle
- crispr
- 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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- 239000002105 nanoparticle Substances 0.000 claims 41
- 238000010453 CRISPR/Cas method Methods 0.000 claims 16
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 15
- 230000008685 targeting Effects 0.000 claims 15
- 210000004027 cell Anatomy 0.000 claims 14
- 239000003814 drug Substances 0.000 claims 14
- 239000003446 ligand Substances 0.000 claims 12
- 239000000203 mixture Substances 0.000 claims 11
- 108020004707 nucleic acids Proteins 0.000 claims 11
- 102000039446 nucleic acids Human genes 0.000 claims 11
- 150000007523 nucleic acids Chemical class 0.000 claims 11
- 108090000765 processed proteins & peptides Proteins 0.000 claims 11
- -1 anionic amino acids Chemical class 0.000 claims 10
- 229920001184 polypeptide Polymers 0.000 claims 10
- 102000004196 processed proteins & peptides Human genes 0.000 claims 10
- 229920000642 polymer Polymers 0.000 claims 9
- 108020004414 DNA Proteins 0.000 claims 8
- 108020005004 Guide RNA Proteins 0.000 claims 8
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims 8
- 230000001939 inductive effect Effects 0.000 claims 7
- 101710185494 Zinc finger protein Proteins 0.000 claims 6
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims 6
- 210000000130 stem cell Anatomy 0.000 claims 6
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 5
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 5
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims 5
- 150000001413 amino acids Chemical group 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 5
- 102000053602 DNA Human genes 0.000 claims 4
- 210000001185 bone marrow Anatomy 0.000 claims 4
- 229920006317 cationic polymer Polymers 0.000 claims 4
- 210000003819 peripheral blood mononuclear cell Anatomy 0.000 claims 4
- 108091005461 Nucleic proteins Proteins 0.000 claims 3
- 125000002091 cationic group Chemical group 0.000 claims 3
- 210000003743 erythrocyte Anatomy 0.000 claims 3
- 238000009472 formulation Methods 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 210000002237 B-cell of pancreatic islet Anatomy 0.000 claims 2
- 108700006830 Drosophila Antp Proteins 0.000 claims 2
- 101000741445 Homo sapiens Calcitonin Proteins 0.000 claims 2
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 claims 2
- CSNNHWWHGAXBCP-UHFFFAOYSA-L Magnesium sulfate Chemical compound [Mg+2].[O-][S+2]([O-])([O-])[O-] CSNNHWWHGAXBCP-UHFFFAOYSA-L 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 2
- 102100027720 SH2 domain-containing protein 1A Human genes 0.000 claims 2
- 101710097989 SH2 domain-containing protein 1A Proteins 0.000 claims 2
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical group O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 claims 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims 2
- 238000010459 TALEN Methods 0.000 claims 2
- 101710192266 Tegument protein VP22 Proteins 0.000 claims 2
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 2
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 2
- 210000001789 adipocyte Anatomy 0.000 claims 2
- 229920006318 anionic polymer Polymers 0.000 claims 2
- 210000001130 astrocyte Anatomy 0.000 claims 2
- 210000003719 b-lymphocyte Anatomy 0.000 claims 2
- 210000003651 basophil Anatomy 0.000 claims 2
- 210000002798 bone marrow cell Anatomy 0.000 claims 2
- OSGAYBCDTDRGGQ-UHFFFAOYSA-L calcium sulfate Chemical compound [Ca+2].[O-]S([O-])(=O)=O OSGAYBCDTDRGGQ-UHFFFAOYSA-L 0.000 claims 2
- 210000004413 cardiac myocyte Anatomy 0.000 claims 2
- 210000001072 colon Anatomy 0.000 claims 2
- 210000004443 dendritic cell Anatomy 0.000 claims 2
- 210000002889 endothelial cell Anatomy 0.000 claims 2
- 210000003979 eosinophil Anatomy 0.000 claims 2
- 210000003714 granulocyte Anatomy 0.000 claims 2
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 210000003630 histaminocyte Anatomy 0.000 claims 2
- 230000000968 intestinal effect Effects 0.000 claims 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- 230000007774 longterm Effects 0.000 claims 2
- 210000003738 lymphoid progenitor cell Anatomy 0.000 claims 2
- 210000002540 macrophage Anatomy 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 210000003593 megakaryocyte Anatomy 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 210000001616 monocyte Anatomy 0.000 claims 2
- 210000000663 muscle cell Anatomy 0.000 claims 2
- 210000000581 natural killer T-cell Anatomy 0.000 claims 2
- 210000000822 natural killer cell Anatomy 0.000 claims 2
- 210000002569 neuron Anatomy 0.000 claims 2
- 210000000440 neutrophil Anatomy 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 210000002363 skeletal muscle cell Anatomy 0.000 claims 2
- 238000010361 transduction Methods 0.000 claims 2
- 230000026683 transduction Effects 0.000 claims 2
- YPFNIPKMNMDDDB-UHFFFAOYSA-K 2-[2-[bis(carboxylatomethyl)amino]ethyl-(2-hydroxyethyl)amino]acetate;iron(3+) Chemical compound [Fe+3].OCCN(CC([O-])=O)CCN(CC([O-])=O)CC([O-])=O YPFNIPKMNMDDDB-UHFFFAOYSA-K 0.000 claims 1
- 102100027207 CD27 antigen Human genes 0.000 claims 1
- 102100025221 CD70 antigen Human genes 0.000 claims 1
- OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 108010033040 Histones Proteins 0.000 claims 1
- 101000914511 Homo sapiens CD27 antigen Proteins 0.000 claims 1
- 101000934356 Homo sapiens CD70 antigen Proteins 0.000 claims 1
- 101000633780 Homo sapiens Signaling lymphocytic activation molecule Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- WHXSMMKQMYFTQS-UHFFFAOYSA-N Lithium Chemical compound [Li] WHXSMMKQMYFTQS-UHFFFAOYSA-N 0.000 claims 1
- FYYHWMGAXLPEAU-UHFFFAOYSA-N Magnesium Chemical compound [Mg] FYYHWMGAXLPEAU-UHFFFAOYSA-N 0.000 claims 1
- 108010052285 Membrane Proteins Proteins 0.000 claims 1
- 102000018697 Membrane Proteins Human genes 0.000 claims 1
- 206010061902 Pancreatic neoplasm Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 108010043958 Peptoids Proteins 0.000 claims 1
- 102000016971 Proto-Oncogene Proteins c-kit Human genes 0.000 claims 1
- 108010014608 Proto-Oncogene Proteins c-kit Proteins 0.000 claims 1
- 102100029215 Signaling lymphocytic activation molecule Human genes 0.000 claims 1
- 101800000868 Tail peptide Proteins 0.000 claims 1
- 102400001102 Tail peptide Human genes 0.000 claims 1
- 208000027276 Von Willebrand disease Diseases 0.000 claims 1
- 229910000147 aluminium phosphate Inorganic materials 0.000 claims 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 1
- 239000011575 calcium Substances 0.000 claims 1
- 229910052791 calcium Inorganic materials 0.000 claims 1
- 239000001506 calcium phosphate Substances 0.000 claims 1
- 229910000389 calcium phosphate Inorganic materials 0.000 claims 1
- 235000011010 calcium phosphates Nutrition 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 230000000925 erythroid effect Effects 0.000 claims 1
- 230000002496 gastric effect Effects 0.000 claims 1
- 238000010362 genome editing Methods 0.000 claims 1
- 108010038082 heparin proteoglycan Proteins 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- CPSYWNLKRDURMG-UHFFFAOYSA-L hydron;manganese(2+);phosphate Chemical compound [Mn+2].OP([O-])([O-])=O CPSYWNLKRDURMG-UHFFFAOYSA-L 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 229910052742 iron Inorganic materials 0.000 claims 1
- 229910000358 iron sulfate Inorganic materials 0.000 claims 1
- BAUYGSIQEAFULO-UHFFFAOYSA-L iron(2+) sulfate (anhydrous) Chemical compound [Fe+2].[O-]S([O-])(=O)=O BAUYGSIQEAFULO-UHFFFAOYSA-L 0.000 claims 1
- 229910052744 lithium Inorganic materials 0.000 claims 1
- 229910001386 lithium phosphate Inorganic materials 0.000 claims 1
- INHCSSUBVCNVSK-UHFFFAOYSA-L lithium sulfate Inorganic materials [Li+].[Li+].[O-]S([O-])(=O)=O INHCSSUBVCNVSK-UHFFFAOYSA-L 0.000 claims 1
- 239000011777 magnesium Substances 0.000 claims 1
- 229910052749 magnesium Inorganic materials 0.000 claims 1
- 235000001055 magnesium Nutrition 0.000 claims 1
- GVALZJMUIHGIMD-UHFFFAOYSA-H magnesium phosphate Chemical compound [Mg+2].[Mg+2].[Mg+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O GVALZJMUIHGIMD-UHFFFAOYSA-H 0.000 claims 1
- 239000004137 magnesium phosphate Substances 0.000 claims 1
- 229910000157 magnesium phosphate Inorganic materials 0.000 claims 1
- 229960002261 magnesium phosphate Drugs 0.000 claims 1
- 235000010994 magnesium phosphates Nutrition 0.000 claims 1
- 229910052943 magnesium sulfate Inorganic materials 0.000 claims 1
- 235000019341 magnesium sulphate Nutrition 0.000 claims 1
- 208000015486 malignant pancreatic neoplasm Diseases 0.000 claims 1
- 229940099596 manganese sulfate Drugs 0.000 claims 1
- 239000011702 manganese sulphate Substances 0.000 claims 1
- 235000007079 manganese sulphate Nutrition 0.000 claims 1
- WPBNNNQJVZRUHP-UHFFFAOYSA-L manganese(2+);methyl n-[[2-(methoxycarbonylcarbamothioylamino)phenyl]carbamothioyl]carbamate;n-[2-(sulfidocarbothioylamino)ethyl]carbamodithioate Chemical compound [Mn+2].[S-]C(=S)NCCNC([S-])=S.COC(=O)NC(=S)NC1=CC=CC=C1NC(=S)NC(=O)OC WPBNNNQJVZRUHP-UHFFFAOYSA-L 0.000 claims 1
- SQQMAOCOWKFBNP-UHFFFAOYSA-L manganese(II) sulfate Chemical compound [Mn+2].[O-]S([O-])(=O)=O SQQMAOCOWKFBNP-UHFFFAOYSA-L 0.000 claims 1
- 201000002528 pancreatic cancer Diseases 0.000 claims 1
- 208000008443 pancreatic carcinoma Diseases 0.000 claims 1
- 229920000744 poly(arginines) Polymers 0.000 claims 1
- 108010077051 polycysteine Proteins 0.000 claims 1
- 239000000377 silicon dioxide Substances 0.000 claims 1
- 210000002784 stomach Anatomy 0.000 claims 1
- RBTVSNLYYIMMKS-UHFFFAOYSA-N tert-butyl 3-aminoazetidine-1-carboxylate;hydrochloride Chemical compound Cl.CC(C)(C)OC(=O)N1CC(N)C1 RBTVSNLYYIMMKS-UHFFFAOYSA-N 0.000 claims 1
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 claims 1
- TWQULNDIKKJZPH-UHFFFAOYSA-K trilithium;phosphate Chemical compound [Li+].[Li+].[Li+].[O-]P([O-])([O-])=O TWQULNDIKKJZPH-UHFFFAOYSA-K 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 208000012137 von Willebrand disease (hereditary or acquired) Diseases 0.000 claims 1
Applications Claiming Priority (10)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662434344P | 2016-12-14 | 2016-12-14 | |
| US62/434,344 | 2016-12-14 | ||
| US201762443522P | 2017-01-06 | 2017-01-06 | |
| US201762443567P | 2017-01-06 | 2017-01-06 | |
| US62/443,522 | 2017-01-06 | ||
| US62/443,567 | 2017-01-06 | ||
| US201762517346P | 2017-06-09 | 2017-06-09 | |
| US62/517,346 | 2017-06-09 | ||
| PCT/US2017/066545 WO2018112282A1 (en) | 2016-12-14 | 2017-12-14 | Compositions and methods for nucleic acid and/or protein payload delivery |
| JP2019531758A JP2020502124A (ja) | 2016-12-14 | 2017-12-14 | 核酸および／またはタンパク質ペイロード送達のための組成物および方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019531758A Division JP2020502124A (ja) | 2016-12-14 | 2017-12-14 | 核酸および／またはタンパク質ペイロード送達のための組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023071853A JP2023071853A (ja) | 2023-05-23 |
| JP2023071853A5 true JP2023071853A5 (enExample) | 2023-10-05 |
Family
ID=62488586
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019531743A Active JP7317706B2 (ja) | 2016-12-14 | 2017-12-14 | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
| JP2019531758A Pending JP2020502124A (ja) | 2016-12-14 | 2017-12-14 | 核酸および／またはタンパク質ペイロード送達のための組成物および方法 |
| JP2023031534A Pending JP2023071853A (ja) | 2016-12-14 | 2023-03-02 | 核酸および／またはタンパク質ペイロード送達のための組成物および方法 |
| JP2023117292A Pending JP2023134730A (ja) | 2016-12-14 | 2023-07-19 | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019531743A Active JP7317706B2 (ja) | 2016-12-14 | 2017-12-14 | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
| JP2019531758A Pending JP2020502124A (ja) | 2016-12-14 | 2017-12-14 | 核酸および／またはタンパク質ペイロード送達のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023117292A Pending JP2023134730A (ja) | 2016-12-14 | 2023-07-19 | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (5) | US20180179553A1 (enExample) |
| EP (2) | EP3554555A4 (enExample) |
| JP (4) | JP7317706B2 (enExample) |
| KR (4) | KR20210035022A (enExample) |
| CN (2) | CN110582301A (enExample) |
| AU (2) | AU2017378427A1 (enExample) |
| CA (2) | CA3045134A1 (enExample) |
| IL (2) | IL267033B2 (enExample) |
| RU (2) | RU2019121801A (enExample) |
| WO (2) | WO2018112282A1 (enExample) |
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| US20210069016A1 (en) * | 2008-11-13 | 2021-03-11 | Gholam A. Peyman | Neurodegenerative Disorder Treatment Method |
| KR102712656B1 (ko) | 2017-01-23 | 2024-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| US11479802B2 (en) | 2017-04-11 | 2022-10-25 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family |
| KR102720855B1 (ko) | 2017-06-15 | 2024-10-25 | 더 리전트 오브 더 유니버시티 오브 캘리포니아 | 표적화된 비-바이러스 dna 삽입 |
| KR20200033259A (ko) | 2017-07-31 | 2020-03-27 | 리제너론 파마슈티칼스 인코포레이티드 | 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물 |
| KR102780441B1 (ko) | 2017-07-31 | 2025-03-17 | 리제너론 파마슈티칼스 인코포레이티드 | Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도 |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| KR102503130B1 (ko) | 2017-10-27 | 2023-02-24 | 더 리전트 오브 더 유니버시티 오브 캘리포니아 | 내인성 t 세포 수용체의 표적화된 대체 |
| CN111885915B (zh) | 2018-03-19 | 2023-04-28 | 瑞泽恩制药公司 | 使用crispr/cas系统对动物进行转录调制 |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| EP3796894A4 (en) * | 2018-04-24 | 2022-05-04 | Ligandal, Inc. | METHODS AND COMPOSITIONS FOR GENOMIC EDITING |
| US20210317436A1 (en) * | 2018-09-08 | 2021-10-14 | Blueallele, Llc | Methods and compositions for modifying the von willebrand factor gene |
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