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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- Japan
- 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 | ||
| 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 |
| PCT/US2017/066545 WO2018112282A1 (en) | 2016-12-14 | 2017-12-14 | Compositions and methods for nucleic acid and/or protein payload delivery |
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 (https=) | 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) | US10975388B2 (https=) |
| EP (2) | EP3554546A4 (https=) |
| JP (4) | JP7317706B2 (https=) |
| KR (4) | KR20240172763A (https=) |
| CN (2) | CN110582302A (https=) |
| AU (2) | AU2017378427A1 (https=) |
| CA (2) | CA3045134A1 (https=) |
| IL (2) | IL267034B2 (https=) |
| RU (2) | RU2019121801A (https=) |
| WO (2) | WO2018112282A1 (https=) |
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| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| WO2018232356A1 (en) | 2017-06-15 | 2018-12-20 | The Regents Of The University Of California | Targeted non-viral dna insertions |
| 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 |
| CN110891420B (zh) | 2017-07-31 | 2022-06-03 | 瑞泽恩制药公司 | Cas转基因小鼠胚胎干细胞和小鼠及其应用 |
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