JP2020502124A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020502124A5 JP2020502124A5 JP2019531758A JP2019531758A JP2020502124A5 JP 2020502124 A5 JP2020502124 A5 JP 2020502124A5 JP 2019531758 A JP2019531758 A JP 2019531758A JP 2019531758 A JP2019531758 A JP 2019531758A JP 2020502124 A5 JP2020502124 A5 JP 2020502124A5
- Authority
- JP
- Japan
- Prior art keywords
- polymer
- isomer
- cationic
- nanoparticles
- amino acids
- 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.)
- Pending
Links
- 239000002105 nanoparticle Substances 0.000 claims description 57
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 44
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 30
- 239000003446 ligand Substances 0.000 claims description 27
- 229920000642 polymer Polymers 0.000 claims description 27
- 230000008685 targeting Effects 0.000 claims description 26
- -1 anionic amino acid Chemical class 0.000 claims description 24
- 239000012634 fragment Substances 0.000 claims description 23
- 125000002091 cationic group Chemical group 0.000 claims description 22
- 229920001184 polypeptide Polymers 0.000 claims description 22
- 239000000203 mixture Substances 0.000 claims description 18
- 238000009833 condensation Methods 0.000 claims description 15
- 230000005494 condensation Effects 0.000 claims description 15
- 108020004707 nucleic acids Proteins 0.000 claims description 13
- 102000039446 nucleic acids Human genes 0.000 claims description 13
- 150000007523 nucleic acids Chemical class 0.000 claims description 13
- 235000018102 proteins Nutrition 0.000 claims description 11
- 102000004169 proteins and genes Human genes 0.000 claims description 11
- 108010033040 Histones Proteins 0.000 claims description 10
- 229920006317 cationic polymer Polymers 0.000 claims description 9
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 8
- 150000001413 amino acids Chemical group 0.000 claims description 7
- 229920006318 anionic polymer Polymers 0.000 claims description 7
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
- 210000000130 stem cell Anatomy 0.000 claims description 4
- 108010024212 E-Selectin Proteins 0.000 claims description 2
- 150000001450 anions Chemical class 0.000 claims description 2
- 150000001768 cations Chemical class 0.000 claims description 2
- 108091005461 Nucleic proteins Proteins 0.000 claims 5
- 102100027720 SH2 domain-containing protein 1A Human genes 0.000 claims 4
- 101710097989 SH2 domain-containing protein 1A Proteins 0.000 claims 4
- 102100025221 CD70 antigen Human genes 0.000 claims 2
- 101000934356 Homo sapiens CD70 antigen Proteins 0.000 claims 2
- 102000008166 Member 25 Tumor Necrosis Factor Receptors Human genes 0.000 claims 2
- 108010060408 Member 25 Tumor Necrosis Factor Receptors Proteins 0.000 claims 2
- 239000012581 transferrin Substances 0.000 claims 2
- WWUZIQQURGPMPG-UHFFFAOYSA-N (-)-D-erythro-Sphingosine Natural products CCCCCCCCCCCCCC=CC(O)C(N)CO WWUZIQQURGPMPG-UHFFFAOYSA-N 0.000 claims 1
- 229940122361 Bisphosphonate Drugs 0.000 claims 1
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 claims 1
- 102100032912 CD44 antigen Human genes 0.000 claims 1
- 208000010772 Dog disease Diseases 0.000 claims 1
- 108700006830 Drosophila Antp Proteins 0.000 claims 1
- 102100023471 E-selectin Human genes 0.000 claims 1
- 102100025101 GATA-type zinc finger protein 1 Human genes 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 102100034223 Golgi apparatus protein 1 Human genes 0.000 claims 1
- 101710087641 Golgi apparatus protein 1 Proteins 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 108091016366 Histone-lysine N-methyltransferase EHMT1 Proteins 0.000 claims 1
- 101000868273 Homo sapiens CD44 antigen Proteins 0.000 claims 1
- 101000741445 Homo sapiens Calcitonin Proteins 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
- 101000800116 Homo sapiens Thy-1 membrane glycoprotein Proteins 0.000 claims 1
- 108010092694 L-Selectin Proteins 0.000 claims 1
- 102100033467 L-selectin Human genes 0.000 claims 1
- 102000010445 Lactoferrin Human genes 0.000 claims 1
- 108010063045 Lactoferrin Proteins 0.000 claims 1
- 108010009254 Lysosomal-Associated Membrane Protein 1 Proteins 0.000 claims 1
- 108010009491 Lysosomal-Associated Membrane Protein 2 Proteins 0.000 claims 1
- 102100035133 Lysosome-associated membrane glycoprotein 1 Human genes 0.000 claims 1
- 102100038225 Lysosome-associated membrane glycoprotein 2 Human genes 0.000 claims 1
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 claims 1
- ZQQLMECVOXKFJK-NXCSZAMKSA-N N-octadecanoylsphingosine 1-phosphate Chemical compound CCCCCCCCCCCCCCCCCC(=O)N[C@@H](COP(O)(O)=O)[C@H](O)\C=C\CCCCCCCCCCCCC ZQQLMECVOXKFJK-NXCSZAMKSA-N 0.000 claims 1
- 108010035766 P-Selectin Proteins 0.000 claims 1
- 102100023472 P-selectin Human genes 0.000 claims 1
- KDYFGRWQOYBRFD-UHFFFAOYSA-N Succinic acid Natural products OC(=O)CCC(O)=O KDYFGRWQOYBRFD-UHFFFAOYSA-N 0.000 claims 1
- 102400001102 Tail peptide Human genes 0.000 claims 1
- 101800000868 Tail peptide Proteins 0.000 claims 1
- 101710192266 Tegument protein VP22 Proteins 0.000 claims 1
- 102100033523 Thy-1 membrane glycoprotein Human genes 0.000 claims 1
- 102000004338 Transferrin Human genes 0.000 claims 1
- 108090000901 Transferrin Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 230000030833 cell death Effects 0.000 claims 1
- 229940106189 ceramide Drugs 0.000 claims 1
- ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- CSSYQJWUGATIHM-IKGCZBKSSA-N l-phenylalanyl-l-lysyl-l-cysteinyl-l-arginyl-l-arginyl-l-tryptophyl-l-glutaminyl-l-tryptophyl-l-arginyl-l-methionyl-l-lysyl-l-lysyl-l-leucylglycyl-l-alanyl-l-prolyl-l-seryl-l-isoleucyl-l-threonyl-l-cysteinyl-l-valyl-l-arginyl-l-arginyl-l-alanyl-l-phenylal Chemical compound C([C@H](N)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CS)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC(C)C)C(=O)NCC(=O)N[C@@H](C)C(=O)N1CCC[C@H]1C(=O)N[C@@H](CO)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CS)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](CC=1C=CC=CC=1)C(O)=O)C1=CC=CC=C1 CSSYQJWUGATIHM-IKGCZBKSSA-N 0.000 claims 1
- 229940078795 lactoferrin Drugs 0.000 claims 1
- 235000021242 lactoferrin Nutrition 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 claims 1
- WWUZIQQURGPMPG-KRWOKUGFSA-N sphingosine Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)CO WWUZIQQURGPMPG-KRWOKUGFSA-N 0.000 claims 1
- DUYSYHSSBDVJSM-KRWOKUGFSA-N sphingosine 1-phosphate Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)COP(O)(O)=O DUYSYHSSBDVJSM-KRWOKUGFSA-N 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 18
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 13
- 239000010931 gold Substances 0.000 description 13
- 229910052737 gold Inorganic materials 0.000 description 13
- 108020005004 Guide RNA Proteins 0.000 description 11
- 108091093037 Peptide nucleic acid Proteins 0.000 description 10
- 238000003556 assay Methods 0.000 description 10
- 230000007717 exclusion Effects 0.000 description 10
- 102000005962 receptors Human genes 0.000 description 10
- 108020003175 receptors Proteins 0.000 description 10
- 210000004369 blood Anatomy 0.000 description 8
- 239000008280 blood Substances 0.000 description 8
- 108020004999 messenger RNA Proteins 0.000 description 8
- 239000004475 Arginine Substances 0.000 description 7
- 108091033409 CRISPR Proteins 0.000 description 7
- 235000009697 arginine Nutrition 0.000 description 7
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 7
- 239000002245 particle Substances 0.000 description 7
- 235000001014 amino acid Nutrition 0.000 description 6
- 108090000623 proteins and genes Proteins 0.000 description 6
- FUOOLUPWFVMBKG-UHFFFAOYSA-N 2-Aminoisobutyric acid Chemical group CC(C)(N)C(O)=O FUOOLUPWFVMBKG-UHFFFAOYSA-N 0.000 description 5
- 238000010354 CRISPR gene editing Methods 0.000 description 5
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 5
- 210000004027 cell Anatomy 0.000 description 5
- 238000009877 rendering Methods 0.000 description 5
- 102000017420 CD3 protein, epsilon/gamma/delta subunit Human genes 0.000 description 4
- 102000006947 Histones Human genes 0.000 description 4
- 102000000588 Interleukin-2 Human genes 0.000 description 4
- 108010002350 Interleukin-2 Proteins 0.000 description 4
- SQVRNKJHWKZAKO-PFQGKNLYSA-N N-acetyl-beta-neuraminic acid Chemical compound CC(=O)N[C@@H]1[C@@H](O)C[C@@](O)(C(O)=O)O[C@H]1[C@H](O)[C@H](O)CO SQVRNKJHWKZAKO-PFQGKNLYSA-N 0.000 description 4
- 102000001558 Sialic Acid Binding Ig-like Lectin 1 Human genes 0.000 description 4
- 108010029176 Sialic Acid Binding Ig-like Lectin 1 Proteins 0.000 description 4
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 description 4
- 150000001412 amines Chemical class 0.000 description 4
- 229940024606 amino acid Drugs 0.000 description 4
- SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 description 4
- 239000011248 coating agent Substances 0.000 description 4
- 238000000576 coating method Methods 0.000 description 4
- 238000009510 drug design Methods 0.000 description 4
- 230000000694 effects Effects 0.000 description 4
- 210000001163 endosome Anatomy 0.000 description 4
- 108010038453 Interleukin-2 Receptors Proteins 0.000 description 3
- 102000010789 Interleukin-2 Receptors Human genes 0.000 description 3
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 description 3
- 239000004472 Lysine Substances 0.000 description 3
- 108020004459 Small interfering RNA Proteins 0.000 description 3
- 238000002983 circular dichroism Methods 0.000 description 3
- 125000001165 hydrophobic group Chemical group 0.000 description 3
- 230000002687 intercalation Effects 0.000 description 3
- 238000009830 intercalation Methods 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 108010011110 polyarginine Proteins 0.000 description 3
- 229920002643 polyglutamic acid Polymers 0.000 description 3
- IGAZHQIYONOHQN-UHFFFAOYSA-N Alexa Fluor 555 Chemical compound C=12C=CC(=N)C(S(O)(=O)=O)=C2OC2=C(S(O)(=O)=O)C(N)=CC=C2C=1C1=CC=C(C(O)=O)C=C1C(O)=O IGAZHQIYONOHQN-UHFFFAOYSA-N 0.000 description 2
- 108010011459 Exenatide Proteins 0.000 description 2
- 101001055144 Homo sapiens Interleukin-2 receptor subunit alpha Proteins 0.000 description 2
- 102100026878 Interleukin-2 receptor subunit alpha Human genes 0.000 description 2
- 101900083372 Rabies virus Glycoprotein Proteins 0.000 description 2
- 230000021736 acetylation Effects 0.000 description 2
- 238000006640 acetylation reaction Methods 0.000 description 2
- 125000000129 anionic group Chemical group 0.000 description 2
- JUFFVKRROAPVBI-PVOYSMBESA-N chembl1210015 Chemical group C([C@@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC(=O)N[C@H]1[C@@H]([C@@H](O)[C@H](O[C@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO[C@]3(O[C@@H](C[C@H](O)[C@H](O)CO)[C@H](NC(C)=O)[C@@H](O)C3)C(O)=O)O2)O)[C@@H](CO)O1)NC(C)=O)C(=O)NCC(=O)NCC(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CO)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](C)C(=O)N1[C@@H](CCC1)C(=O)N1[C@@H](CCC1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CO)C(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@@H](NC(=O)[C@H](C)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCSC)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CO)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CO)NC(=O)[C@@H](NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@@H](NC(=O)CNC(=O)[C@H](CCC(O)=O)NC(=O)CNC(=O)[C@@H](N)CC=1NC=NC=1)[C@@H](C)O)[C@@H](C)O)C(C)C)C1=CC=CC=C1 JUFFVKRROAPVBI-PVOYSMBESA-N 0.000 description 2
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 description 2
- 238000010586 diagram Methods 0.000 description 2
- 229960001519 exenatide Drugs 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 description 2
- 238000002952 image-based readout Methods 0.000 description 2
- 238000003780 insertion Methods 0.000 description 2
- 230000037431 insertion Effects 0.000 description 2
- 230000003993 interaction Effects 0.000 description 2
- 210000003819 peripheral blood mononuclear cell Anatomy 0.000 description 2
- 230000006337 proteolytic cleavage Effects 0.000 description 2
- 102220328583 rs111822347 Human genes 0.000 description 2
- 239000000377 silicon dioxide Substances 0.000 description 2
- 238000004088 simulation Methods 0.000 description 2
- 238000006467 substitution reaction Methods 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- 101710150620 Anionic peptide Proteins 0.000 description 1
- 102000004225 Cathepsin B Human genes 0.000 description 1
- 108090000712 Cathepsin B Proteins 0.000 description 1
- 102000003908 Cathepsin D Human genes 0.000 description 1
- 108090000258 Cathepsin D Proteins 0.000 description 1
- ZEOWTGPWHLSLOG-UHFFFAOYSA-N Cc1ccc(cc1-c1ccc2c(n[nH]c2c1)-c1cnn(c1)C1CC1)C(=O)Nc1cccc(c1)C(F)(F)F Chemical compound Cc1ccc(cc1-c1ccc2c(n[nH]c2c1)-c1cnn(c1)C1CC1)C(=O)Nc1cccc(c1)C(F)(F)F ZEOWTGPWHLSLOG-UHFFFAOYSA-N 0.000 description 1
- 102000015689 E-Selectin Human genes 0.000 description 1
- 102100023593 Fibroblast growth factor receptor 1 Human genes 0.000 description 1
- 101710182386 Fibroblast growth factor receptor 1 Proteins 0.000 description 1
- 102000003688 G-Protein-Coupled Receptors Human genes 0.000 description 1
- 108090000045 G-Protein-Coupled Receptors Proteins 0.000 description 1
- 102000017286 Histone H2A Human genes 0.000 description 1
- 108050005231 Histone H2A Proteins 0.000 description 1
- 101100220044 Homo sapiens CD34 gene Proteins 0.000 description 1
- 101000738771 Homo sapiens Receptor-type tyrosine-protein phosphatase C Proteins 0.000 description 1
- 102000008394 Immunoglobulin Fragments Human genes 0.000 description 1
- 108010021625 Immunoglobulin Fragments Proteins 0.000 description 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 description 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 description 1
- 108010090054 Membrane Glycoproteins Proteins 0.000 description 1
- 102000012750 Membrane Glycoproteins Human genes 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 240000007019 Oxalis corniculata Species 0.000 description 1
- 102000007327 Protamines Human genes 0.000 description 1
- 108010007568 Protamines Proteins 0.000 description 1
- 108010076504 Protein Sorting Signals Proteins 0.000 description 1
- 102000016971 Proto-Oncogene Proteins c-kit Human genes 0.000 description 1
- 108010014608 Proto-Oncogene Proteins c-kit Proteins 0.000 description 1
- 102100037422 Receptor-type tyrosine-protein phosphatase C Human genes 0.000 description 1
- 102000007073 Sialic Acid Binding Immunoglobulin-like Lectins Human genes 0.000 description 1
- 108010047827 Sialic Acid Binding Immunoglobulin-like Lectins Proteins 0.000 description 1
- 230000010933 acylation Effects 0.000 description 1
- 238000005917 acylation reaction Methods 0.000 description 1
- 230000003281 allosteric effect Effects 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 210000002798 bone marrow cell Anatomy 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 238000004113 cell culture Methods 0.000 description 1
- 230000024245 cell differentiation Effects 0.000 description 1
- 230000004663 cell proliferation Effects 0.000 description 1
- 230000008045 co-localization Effects 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 238000003271 compound fluorescence assay Methods 0.000 description 1
- 238000004132 cross linking Methods 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 125000002228 disulfide group Chemical group 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 230000012202 endocytosis Effects 0.000 description 1
- 210000002889 endothelial cell Anatomy 0.000 description 1
- 230000003511 endothelial effect Effects 0.000 description 1
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- MHMNJMPURVTYEJ-UHFFFAOYSA-N fluorescein-5-isothiocyanate Chemical compound O1C(=O)C2=CC(N=C=S)=CC=C2C21C1=CC=C(O)C=C1OC1=CC(O)=CC=C21 MHMNJMPURVTYEJ-UHFFFAOYSA-N 0.000 description 1
- 230000013595 glycosylation Effects 0.000 description 1
- 238000006206 glycosylation reaction Methods 0.000 description 1
- 229960002897 heparin Drugs 0.000 description 1
- 229920000669 heparin Polymers 0.000 description 1
- 125000000487 histidyl group Chemical group [H]N([H])C(C(=O)O*)C([H])([H])C1=C([H])N([H])C([H])=N1 0.000 description 1
- 238000003384 imaging method Methods 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 108091070501 miRNA Proteins 0.000 description 1
- 239000002679 microRNA Substances 0.000 description 1
- 230000003278 mimic effect Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 239000013642 negative control Substances 0.000 description 1
- 210000001178 neural stem cell Anatomy 0.000 description 1
- 230000007935 neutral effect Effects 0.000 description 1
- 238000010899 nucleation Methods 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 229940048914 protamine Drugs 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 229920005989 resin Polymers 0.000 description 1
- 239000011347 resin Substances 0.000 description 1
- 238000005096 rolling process Methods 0.000 description 1
- 230000011664 signaling Effects 0.000 description 1
- 230000006641 stabilisation Effects 0.000 description 1
- 238000011105 stabilization Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 230000007704 transition Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023031534A 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 |
Applications Claiming Priority (9)
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023031534A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020502124A JP2020502124A (ja) | 2020-01-23 |
| JP2020502124A5 true JP2020502124A5 (https=) | 2021-08-12 |
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 (1)
| 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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
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=) |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20210069016A1 (en) * | 2008-11-13 | 2021-03-11 | Gholam A. Peyman | Neurodegenerative Disorder Treatment Method |
| 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 |
| 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 |
| JP7101419B2 (ja) | 2017-10-27 | 2022-07-15 | 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 | 内因性ｔ細胞受容体の標的置換 |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| KR20210039983A (ko) * | 2018-04-24 | 2021-04-12 | 리간달 인코포레이티드 | 게놈 편집을 위한 방법 및 조성물 |
| WO2020051396A1 (en) * | 2018-09-08 | 2020-03-12 | Blueallele, Llc | Methods and compositions for modifying the von willebrand factor gene |
| KR102769565B1 (ko) | 2018-09-19 | 2025-02-20 | 애로우헤드 파마슈티컬스 인코포레이티드 | 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 |
| JP2022512977A (ja) | 2018-11-08 | 2022-02-07 | サメイション バイオ， インコーポレイテッド | ミニヌクレオソームコアタンパク質及び核酸送達における使用 |
| AU2019392748A1 (en) * | 2018-12-05 | 2021-06-10 | Fred Hutchinson Cancer Center | Reduced and minimal manipulation manufacturing of genetically-modified cells |
| CN113544266A (zh) * | 2018-12-17 | 2021-10-22 | 博德研究所 | Crispr相关转座酶系统和其使用方法 |
| US11249941B2 (en) * | 2018-12-21 | 2022-02-15 | Palo Alto Research Center Incorporated | Exabyte-scale data storage using sequence-controlled polymers |
| CN111363013B (zh) * | 2018-12-25 | 2021-11-19 | 深圳先进技术研究院 | 多组分纳米颗粒簇的构建方法 |
| CN109735540B (zh) * | 2019-01-22 | 2022-05-31 | 南京鼓楼医院 | SH2D1A基因、sgRNA及其应用 |
| US20210379192A1 (en) * | 2019-01-25 | 2021-12-09 | Mantra Bio, Inc. | Skeletal muscle targeting moieties and uses thereof |
| AU2020232238B2 (en) | 2019-03-01 | 2025-10-30 | Flagship Pioneering Innovations Vi, Llc | Polyribonucleotides and cosmetic uses thereof |
| MA55082A (fr) | 2019-03-01 | 2022-01-05 | Flagship Pioneering Innovations Vi Llc | Compositions, procédés, et kits pour l'administration de polyribonucléotides |
| CA3138991A1 (en) * | 2019-05-02 | 2020-11-05 | Ligandal, Inc. | Methods and compositions for diagnostically-responsive ligand-targeted delivery of therapeutic agents |
| WO2020261227A1 (en) | 2019-06-26 | 2020-12-30 | Biorchestra Co., Ltd. | Micellar nanoparticles and uses thereof |
| JP7630904B2 (ja) * | 2019-07-23 | 2025-02-18 | 株式会社東芝 | 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法 |
| CN110628723B (zh) * | 2019-09-05 | 2021-05-04 | 清华大学 | 基因修饰MSCs治疗2型糖尿病 |
| US20210163935A1 (en) * | 2019-09-05 | 2021-06-03 | Trevor P. Castor | Targeted Critical Fluid Nanoparticles Platform for Delivery of Nucleic Acids for Treatment of HIV-1 and Other Diseases |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| CN112779317A (zh) * | 2019-11-11 | 2021-05-11 | 香港中文大学深圳研究院 | 一种用于小分子核糖核酸检测的试剂及其应用 |
| WO2021097305A1 (en) * | 2019-11-15 | 2021-05-20 | The Regents Of The University Of California | Supramolecular nanosubstrate-mediated delivery system enables crispr/cas9 knockin of hemoglobin beta gene-a potential therapeutic solution for hemoglobinopathies |
| CN115698282A (zh) | 2020-01-13 | 2023-02-03 | 福路伦特生物科学公司 | 单细胞测序 |
| WO2021154888A1 (en) | 2020-01-27 | 2021-08-05 | Mantra Bio. Inc. | Non-naturally occurring vesicles comprising a chimeric vesicle localization moiety, methods of making and uses thereof |
| CA3172878A1 (en) * | 2020-02-25 | 2021-09-02 | Ligandal, Inc. | Identification of biomimetic viral peptides and uses thereof |
| WO2021168575A1 (en) * | 2020-02-27 | 2021-09-02 | Quadrumix Biotechnology Inc. | Polypeptides directed against viral infection and uses thereof |
| US11866782B2 (en) | 2020-03-16 | 2024-01-09 | Fluent Biosciences Inc. | Multi-omic analysis in monodisperse droplets |
| WO2021195169A1 (en) | 2020-03-24 | 2021-09-30 | Fluent Biosciences Inc. | Viral detection using template emulsification |
| AU2021254697A1 (en) | 2020-04-13 | 2022-11-10 | Summation Bio Inc. | Modified mini-nucleosome core proteins and use in nucleic acid delivery |
| EP4153223A1 (en) | 2020-05-20 | 2023-03-29 | Flagship Pioneering Innovations VI, LLC | Immunogenic compositions and uses thereof |
| EP4182477A4 (en) | 2020-07-15 | 2024-09-11 | Fluent Biosciences Inc. | MULTI-LEVEL LIGATION OLIGOS |
| CN111968707B (zh) * | 2020-08-07 | 2022-06-17 | 上海交通大学 | 基于能量的原子结构与电子密度图多目标优化拟合预测方法 |
| CN111888333A (zh) * | 2020-08-11 | 2020-11-06 | 深圳大学 | 一种转铁蛋白受体靶向的纳米胶束及其制备方法与应用 |
| WO2022040605A1 (en) * | 2020-08-21 | 2022-02-24 | Ligandal, Inc. | Compositions and methods for nanoparticle seed substrates |
| AU2021336976A1 (en) | 2020-09-03 | 2023-03-23 | Flagship Pioneering Innovations Vi, Llc | Immunogenic compositions and uses thereof |
| JP2024503303A (ja) * | 2020-12-30 | 2024-01-25 | バイオーケストラ カンパニー， リミテッド | ミセルナノ粒子及びその使用 |
| WO2022144811A1 (en) * | 2020-12-30 | 2022-07-07 | Biorchestra Co., Ltd. | Micellar nanoparticles and uses thereof |
| CA3218849A1 (en) * | 2021-05-14 | 2022-11-17 | University Of Rochester | Variants of sirt6 for use in preventing and/or treating age-related diseases |
| CA3220477A1 (en) * | 2021-05-18 | 2022-11-24 | Fluent Biosciences Inc. | Perturbed genomic expression in pretemplated instant partitions |
| US20230076395A1 (en) * | 2021-08-17 | 2023-03-09 | California Institute Of Technology | Cell-to-cell delivery of rna circuits |
| KR102775653B1 (ko) * | 2021-09-15 | 2025-03-05 | 한국기초과학지원연구원 | 아르기닌 리치 펩타이드를 포함하는 단백질 분해제 및 이를 이용한 단백질 이상질환의 예방 또는 치료용 약학적 조성물 |
| JP2024537991A (ja) | 2021-10-14 | 2024-10-18 | アーセナル バイオサイエンシズ インコーポレイテッド | 共発現されるｓｈＲＮＡと論理ゲートシステムとを有する免疫細胞 |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| CN115304756B (zh) * | 2022-01-30 | 2023-05-09 | 上海科技大学 | 一种五元脂质纳米颗粒及其制备方法和应用 |
| CN114480672B (zh) * | 2022-02-22 | 2023-08-15 | 青岛农业大学 | 一种通过miR-145筛选高产肉率碱地黑牛的方法 |
| CN114933658B (zh) * | 2022-04-24 | 2023-07-11 | 深圳市鹏泰生物科技有限公司 | 一种短肽元件及其应用方法 |
| CN115184480B (zh) * | 2022-05-24 | 2024-07-09 | 南京中医药大学 | 一种用于鉴定华蟾素制剂或蟾皮的特征多肽及其含量测定方法与应用 |
| CN115177734B (zh) * | 2022-07-05 | 2025-03-14 | 中国人民解放军空军军医大学 | 一种靶向干预线粒体基因表达的工程化外泌体及其构建方法和应用 |
| CN115350330B (zh) * | 2022-09-01 | 2023-10-20 | 北京化工大学 | 一种负电性小分子调控的表面在蛋白差异性黏附上的应用 |
| US12534721B2 (en) | 2022-10-06 | 2026-01-27 | Illumina, Inc. | Quantitative detection and analysis of moleculesip |
| WO2024098238A1 (en) * | 2022-11-08 | 2024-05-16 | Pulsar Therapeutics Inc. | Methods for treating hemoglobinopathies |
| WO2025049677A1 (en) * | 2023-08-31 | 2025-03-06 | Tufts Medical Center, Inc. | Use of nanoparticles in therapeutic and imaging methods |
| AU2024367190A1 (en) * | 2023-10-23 | 2026-04-23 | The Board Of Regents Of The University Of Texas System | Novel endoplasmic reticulum targeting signal peptides and uses thereof |
| CN117599200A (zh) * | 2023-10-27 | 2024-02-27 | 华中科技大学 | 一种新型的双链rna递送载体及应用 |
| WO2025102075A1 (en) * | 2023-11-09 | 2025-05-15 | The Board Of Regents Of The University Of Texas System | Endogenous synapse targeting signal peptides and uses thereof |
| WO2025207378A1 (en) * | 2024-03-25 | 2025-10-02 | Massachusetts Institute Of Technology | Scalable and high throughput assembly of layer-by-layer nanoparticles |
| KR20250160320A (ko) * | 2024-05-03 | 2025-11-12 | 한양대학교 산학협력단 | 엔도좀 탈출 효율이 향상된 항 인간 트랜스페린 수용체 항체, 및 이를 이용한 다중특이적 항체 및 약학 조성물 |
Family Cites Families (111)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US5837533A (en) | 1994-09-28 | 1998-11-17 | American Home Products Corporation | Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent |
| US6051429A (en) | 1995-06-07 | 2000-04-18 | Life Technologies, Inc. | Peptide-enhanced cationic lipid transfections |
| US6379966B2 (en) | 1999-02-26 | 2002-04-30 | Mirus Corporation | Intravascular delivery of non-viral nucleic acid |
| EP1035158A4 (en) | 1997-10-29 | 2002-01-30 | Kanebo Ltd | RESIN COUPLING WITH BIODEGRADABILITY AND FOAMABILITY |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| CA2372214A1 (en) | 1999-04-30 | 2000-11-09 | Amylin Pharmaceuticals, Inc. | Modified exendins and exendin agonists |
| US7098032B2 (en) | 2001-01-02 | 2006-08-29 | Mirus Bio Corporation | Compositions and methods for drug delivery using pH sensitive molecules |
| AU2001257331A1 (en) | 2000-04-28 | 2001-11-12 | Sangamo Biosciences, Inc. | Methods for designing exogenous regulatory molecules |
| US7355019B2 (en) | 2000-06-06 | 2008-04-08 | Sibtech, Inc. | Cysteine-containing peptide tag for site-specific conjugation of proteins |
| US20030082561A1 (en) | 2000-07-21 | 2003-05-01 | Takashi Sera | Zinc finger domain recognition code and uses thereof |
| ES2260291T3 (es) | 2000-09-28 | 2006-11-01 | Chiron Corporation | Microparticulas para la administracion de acidos nucleicos heterologos. |
| IL155812A0 (en) | 2000-12-07 | 2003-12-23 | Lilly Co Eli | Glp-1 fusion proteins |
| WO2002057293A2 (en) | 2001-01-22 | 2002-07-25 | Sangamo Biosciences, Inc. | Modified zinc finger binding proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| US20040102606A1 (en) | 2001-04-24 | 2004-05-27 | Danuta Balicki | Histone H2A -derived peptides useful in gene delivery |
| US6805904B2 (en) | 2002-02-20 | 2004-10-19 | International Business Machines Corporation | Process of forming a multilayer nanoparticle-containing thin film self-assembly |
| WO2003080809A2 (en) | 2002-03-21 | 2003-10-02 | Sangamo Biosciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| EP1499895B1 (en) | 2002-04-22 | 2008-05-07 | University Of Florida | Functionalized nanoparticles and methods of use |
| EP1519714B1 (en) | 2002-06-28 | 2010-10-20 | Protiva Biotherapeutics Inc. | Method and apparatus for producing liposomes |
| WO2004085998A2 (en) | 2003-03-28 | 2004-10-07 | The Children's Hospital Of Philadelphia | Biomimetic hierarchies using functionalized nanoparticles as building blocks |
| AU2004263865B2 (en) | 2003-08-08 | 2007-05-17 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| JP2007502833A (ja) | 2003-08-21 | 2007-02-15 | サウスウエスト・リサーチ・インスティチュート | 骨格に標的とされるナノ粒子 |
| EP1748796A4 (en) | 2004-03-05 | 2008-08-27 | Univ Texas | MULTILAYER NANOMEDICINE DELIVERY SYSTEM AND METHOD |
| MXPA06012722A (es) | 2004-05-05 | 2007-02-14 | Atugen Ag | Lipidos, complejos de lipido y su uso. |
| WO2005123142A1 (en) | 2004-06-10 | 2005-12-29 | Abraxis Bioscience, Inc. | A method using inorganic nanoparticles as non-viral vectors for gene therapy |
| US20060088599A1 (en) | 2004-08-02 | 2006-04-27 | Prasad Paras N | Amino functionalized ORMOSIL nanoparticles as delivery vehicles |
| US20090220587A1 (en) | 2005-02-01 | 2009-09-03 | United State Army | Liposomal drug delivery constructs targeted by lipid-conjugated peptide ligands |
| WO2007109354A2 (en) | 2006-03-21 | 2007-09-27 | Amylin Pharmaceuticals, Inc. | Peptide-peptidase inhibitor conjugates and methods of using same |
| DK2019683T4 (da) | 2006-04-25 | 2022-08-29 | Univ California | Indgivelse af vækstfaktorer til behandling af CNS-lidelser |
| US7928186B2 (en) * | 2006-08-02 | 2011-04-19 | Phoenix Pharmaceuticals, Inc. | Cell permeable bioactive peptide conjugates |
| BRPI0716427A2 (pt) | 2006-08-11 | 2014-03-11 | Dow Agrosciences Llc | Recombinação homóloga mediada por nuclease de apêndice de zinco |
| CA2689042A1 (en) | 2007-02-16 | 2008-08-28 | Merck & Co., Inc. | Compositions and methods for potentiated activity of biologicaly active molecules |
| US20100196492A1 (en) | 2007-03-08 | 2010-08-05 | Green Jordan J | Electrostatic coating of particles for drug delivery |
| TWI428135B (zh) | 2007-03-26 | 2014-03-01 | Hirofumi Takeuchi | And a carrier composition for quick-acting nucleic acid delivery |
| AU2008305590A1 (en) | 2007-09-27 | 2009-04-02 | Sangamo Therapeutics, Inc. | Genomic editing in zebrafish using zinc finger nucleases |
| EP2205752B1 (en) | 2007-10-25 | 2016-08-10 | Sangamo BioSciences, Inc. | Methods and compositions for targeted integration |
| EP2222283A2 (en) | 2007-10-29 | 2010-09-01 | University of Massachusetts | Yeast cell wall protein (ycwp) encapsulated multilayered nanoparticles for nucleic acid delivery (sirna) |
| US20090155289A1 (en) | 2007-11-01 | 2009-06-18 | Steve Roberts | Furin-cleavable peptide linkers for drug-ligand conjugates |
| US8323618B2 (en) | 2007-11-07 | 2012-12-04 | University Of Houston System | Ultrasmall superparamagnetic iron oxide nanoparticles and uses thereof |
| AU2008325122A1 (en) | 2007-11-09 | 2009-05-14 | Northeastern University | Self-assembling micelle-like nanoparticles for systemic gene delivery |
| GB0725321D0 (en) | 2007-12-31 | 2008-02-06 | Syntaxin Ltd | Delivery vehicles |
| US7999025B2 (en) | 2008-01-28 | 2011-08-16 | University Of Utah Research Foundation | Asymmetrically-functionalized nanoparticles organized on one-dimensional chains |
| US8344116B2 (en) * | 2008-03-17 | 2013-01-01 | Case Western Reserve University | Polymers and complexes for delivery of nucleic acids to intracellular targets |
| US8324333B2 (en) | 2008-06-05 | 2012-12-04 | Wisconsin Alumni Research Foundation | Anionic charge-dynamic polymers for release of cationic agents |
| US8404636B2 (en) | 2008-07-17 | 2013-03-26 | George Mason Intellectual Properties, Inc. | Targeted delivery of antimicrobial agents |
| CA2739608A1 (en) * | 2008-10-07 | 2010-04-15 | The Regents Of The University Of California | Recombinant nell protein production |
| CN111808084A (zh) | 2008-11-10 | 2020-10-23 | 阿布特斯生物制药公司 | 用于递送治疗剂的新型脂质和组合物 |
| EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| TWI576432B (zh) | 2009-04-07 | 2017-04-01 | 陶氏農業科學公司 | 序列特異性核酸酶之奈米粒子媒介式遞送技術 |
| RU2012103240A (ru) | 2009-07-02 | 2013-08-10 | Ангиокем Инк. | Мультимерные пептидные конъюгаты и их применение |
| US20110077581A1 (en) | 2009-09-25 | 2011-03-31 | Georgia Tech Research Corporation | Targeted cellular delivery of nanoparticles |
| CN104147611A (zh) | 2009-09-30 | 2014-11-19 | 葛兰素集团有限公司 | 具有延长的半衰期的药物融合体和缀合物 |
| AU2010305284A1 (en) | 2009-10-06 | 2012-05-03 | Angiochem Inc. | Compositions and methods for the transport of therapeutic agents |
| WO2011072246A2 (en) | 2009-12-10 | 2011-06-16 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| JPWO2011096408A1 (ja) | 2010-02-02 | 2013-06-10 | 国立大学法人 東京大学 | 複合体微粒子およびその製造方法、ならびに該複合体微粒子を用いた薬学組成物 |
| KR101198715B1 (ko) | 2010-05-14 | 2012-11-13 | 한국생명공학연구원 | 핵산 및 친수성 음이온 화합물의 고효율 포획을 위한 비대칭 리포솜 및 이의 제조방법 |
| WO2012012352A2 (en) * | 2010-07-19 | 2012-01-26 | Amidebio, Llc | Modified peptides and proteins |
| US9233163B2 (en) | 2010-12-28 | 2016-01-12 | The Children's Hospital Of Philadelphia | Hydrolytically releasable prodrugs for sustained release nanoparticle formulations |
| US9074187B2 (en) | 2011-03-21 | 2015-07-07 | Board Of Trustees Of The University Of Arkansas | Nanostructural materials that increase mineralization in bone cells and affect gene expression through miRNA regulation and applications of same |
| AU2012236099A1 (en) | 2011-03-31 | 2013-10-03 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| CA3111953C (en) | 2011-04-05 | 2023-10-24 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| AU2012249390B2 (en) | 2011-04-27 | 2017-03-30 | Amyris, Inc. | Methods for genomic modification |
| US8916696B2 (en) | 2011-06-12 | 2014-12-23 | City Of Hope | Aptamer-mRNA conjugates for targeted protein or peptide expression and methods for their use |
| WO2013058812A1 (en) | 2011-10-19 | 2013-04-25 | President And Fellows Of Harvard College | Targeted delivery to pancreatic islet endothelial cells |
| US8450107B1 (en) | 2011-11-30 | 2013-05-28 | The Broad Institute Inc. | Nucleotide-specific recognition sequences for designer TAL effectors |
| CZ201220A3 (cs) | 2012-01-13 | 2013-07-17 | Ústav organické chemie a biochemie Akademie ved CR, v.v.i. | Lipopolyaminy sperminového typu pro konstrukci liposomálních transfekcních systému |
| EP2841056A4 (en) | 2012-04-23 | 2015-09-16 | Massachusetts Inst Technology | STABLE LAYER COATED PARTICLES |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| HRP20181855T1 (hr) | 2012-06-08 | 2018-12-28 | Nitto Denko Corporation | Lipidi za formulacije namijenjene unosu terapijskog sredstva |
| US9993439B2 (en) | 2012-06-20 | 2018-06-12 | University Of Waterloo | Mucoadhesive nanoparticle delivery system |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| IL239344B2 (en) | 2012-12-12 | 2024-06-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US20140186843A1 (en) | 2012-12-12 | 2014-07-03 | Massachusetts Institute Of Technology | Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| CN105074061B (zh) | 2012-12-13 | 2021-03-09 | 美国陶氏益农公司 | 位点特异性核酸酶活性的dna检测方法 |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| US10227610B2 (en) | 2013-02-25 | 2019-03-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing nuclease-mediated gene disruption |
| US9504747B2 (en) | 2013-03-08 | 2016-11-29 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| JP6456853B2 (ja) * | 2013-03-13 | 2019-01-23 | ビオクリネ エービー | 糖尿病を治療および／または発症を制限するための方法 |
| RU2662932C2 (ru) | 2013-03-14 | 2018-07-31 | Карибо Биосайенсиз, Инк. | Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| WO2014144155A1 (en) | 2013-03-15 | 2014-09-18 | Regents Of The University Of Minnesota | Engineering plant genomes using crispr/cas systems |
| US11332719B2 (en) | 2013-03-15 | 2022-05-17 | The Broad Institute, Inc. | Recombinant virus and preparations thereof |
| US20140364333A1 (en) | 2013-03-15 | 2014-12-11 | President And Fellows Of Harvard College | Methods for Live Imaging of Cells |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US20140349400A1 (en) | 2013-03-15 | 2014-11-27 | Massachusetts Institute Of Technology | Programmable Modification of DNA |
| CN105263312A (zh) | 2013-04-05 | 2016-01-20 | 美国陶氏益农公司 | 用于在植物基因组内整合外源序列的方法和组合物 |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| EP2994531B1 (en) | 2013-05-10 | 2018-03-28 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| CA2913234A1 (en) | 2013-05-22 | 2014-11-27 | Northwestern University | Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis |
| US9873907B2 (en) | 2013-05-29 | 2018-01-23 | Agilent Technologies, Inc. | Method for fragmenting genomic DNA using CAS9 |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| EP3049116B1 (en) | 2013-09-23 | 2019-01-02 | Rensselaer Polytechnic Institute | Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations |
| WO2015048577A2 (en) | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| CA2926797A1 (en) | 2013-10-14 | 2015-04-23 | Nanosphere Health Sciences, Llc | Nanoparticle compositions and methods as carriers of nutraceutical factors across cell membranes and biological barriers |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| CN107073028A (zh) | 2013-12-13 | 2017-08-18 | 南洋理工大学 | 多层纳米颗粒及其制作方法和使用方法 |
| US10588972B2 (en) * | 2014-06-02 | 2020-03-17 | Li-Cor, Inc. | Phthalocyanine probes and uses thereof |
| JP6637988B2 (ja) | 2014-11-18 | 2020-01-29 | アークトゥルス セラピューティクス， インコーポレイテッド | Ｒｎａ送達のためのイオン化可能カチオン性脂質 |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
-
2017
- 2017-12-14 AU AU2017378427A patent/AU2017378427A1/en not_active Abandoned
- 2017-12-14 KR KR1020247039185A patent/KR20240172763A/ko active Pending
- 2017-12-14 EP EP17882004.9A patent/EP3554546A4/en not_active Withdrawn
- 2017-12-14 IL IL267034A patent/IL267034B2/en unknown
- 2017-12-14 CN CN201780084854.4A patent/CN110582302A/zh active Pending
- 2017-12-14 JP JP2019531743A patent/JP7317706B2/ja active Active
- 2017-12-14 US US15/842,820 patent/US10975388B2/en active Active
- 2017-12-14 RU RU2019121801A patent/RU2019121801A/ru unknown
- 2017-12-14 IL IL267033A patent/IL267033B2/en unknown
- 2017-12-14 CN CN201780085854.6A patent/CN110582301A/zh active Pending
- 2017-12-14 JP JP2019531758A patent/JP2020502124A/ja active Pending
- 2017-12-14 CA CA3045134A patent/CA3045134A1/en active Pending
- 2017-12-14 WO PCT/US2017/066545 patent/WO2018112282A1/en not_active Ceased
- 2017-12-14 KR KR1020197020171A patent/KR102641844B1/ko active Active
- 2017-12-14 WO PCT/US2017/066541 patent/WO2018112278A1/en not_active Ceased
- 2017-12-14 US US15/842,829 patent/US20180179553A1/en not_active Abandoned
- 2017-12-14 CA CA3045131A patent/CA3045131A1/en not_active Abandoned
- 2017-12-14 RU RU2019121992A patent/RU2019121992A/ru unknown
- 2017-12-14 AU AU2017378431A patent/AU2017378431A1/en not_active Abandoned
- 2017-12-14 KR KR1020247006016A patent/KR20240027888A/ko active Pending
- 2017-12-14 EP EP17881690.6A patent/EP3554555A4/en active Pending
- 2017-12-14 KR KR1020197020175A patent/KR20210035022A/ko not_active Ceased
-
2019
- 2019-12-02 US US16/701,014 patent/US20200095605A1/en active Pending
-
2020
- 2020-11-25 US US17/104,552 patent/US12123012B2/en active Active
-
2023
- 2023-03-02 JP JP2023031534A patent/JP2023071853A/ja active Pending
- 2023-07-19 JP JP2023117292A patent/JP2023134730A/ja active Pending
- 2023-08-29 US US18/458,076 patent/US20240026375A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020502124A5 (https=) | ||
| JP2020502120A5 (https=) | ||
| JP2023071853A (ja) | 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 | |
| JP2023071853A5 (https=) | ||
| Kichler et al. | Cationic amphipathic histidine-rich peptides for gene delivery | |
| EP3049116B1 (en) | Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations | |
| Clinton et al. | Dynamin-related protein 1 oligomerization in solution impairs functional interactions with membrane-anchored mitochondrial fission factor | |
| JP7177047B2 (ja) | ポリペプチドカーゴを標的真核細胞の細胞外間隙からサイトゾルおよび／または核に送達するための合理的に設計された合成ペプチドシャトル剤、その使用、それに関連する方法およびキット | |
| Stahlhut et al. | Identification of filamin as a novel ligand for caveolin-1: evidence for the organization of caveolin-1–associated membrane domains by the actin cytoskeleton | |
| Lees et al. | Structure and function of proteolipids in myelin and non-myelin membranes | |
| JP2019531316A5 (https=) | ||
| JP2021521825A (ja) | ゲノム編集方法及び組成物 | |
| Berezhnoy et al. | Principles of electrostatic interactions and self-assembly in lipid/peptide/DNA systems: applications to gene delivery | |
| CN111647042A (zh) | 骨重建调节多肽及应用 | |
| KR101064914B1 (ko) | 자가면역질환, 알러지성 질환 및 염증성 질환 치료용 약제 조성물 및 이의 전달 방법 | |
| Wang et al. | RSU-1 interaction with prohibitin-2 links cell–extracellular matrix detachment to downregulation of ERK signaling | |
| Bauer et al. | Cellular distribution of innexin 1 and 2 gap junctional channel proteins in epithelia of the Drosophila embryo | |
| US20090253206A1 (en) | Nuclear transport agent and method for producing said agent | |
| Futaki | Design and Creation of Functional Membrane-Interacting Peptides | |
| CN102250222A (zh) | 一种可靶向运输dna到神经细胞及脑部的肽 | |
| Mohan | Regulation of assembly and cell surface dynamics of caveolae | |
| Kharkyanen | Theoretical Investigation of the Feasibility of PTD-Mediated Translocation of Proteins Across Artificial Membranes | |
| Reulen | Multivalent peptides and proteins for collagen imaging | |
| Rodriguez et al. | Nanoparticles Masquerade as “Self” to Inhibit Phagocytosis | |
| Ma et al. | RUNNING TITLES |