CA2373547A1 - Methods for induction of tolerance to adenoviral vectors and transgene products - Google Patents
Methods for induction of tolerance to adenoviral vectors and transgene products Download PDFInfo
- Publication number
- CA2373547A1 CA2373547A1 CA002373547A CA2373547A CA2373547A1 CA 2373547 A1 CA2373547 A1 CA 2373547A1 CA 002373547 A CA002373547 A CA 002373547A CA 2373547 A CA2373547 A CA 2373547A CA 2373547 A1 CA2373547 A1 CA 2373547A1
- Authority
- CA
- Canada
- Prior art keywords
- vector
- transgene
- nucleic acid
- cells
- heterologous nucleic
- 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.)
- Abandoned
Links
- 239000013598 vector Substances 0.000 title claims abstract description 250
- 108700019146 Transgenes Proteins 0.000 title claims abstract description 135
- 238000000034 method Methods 0.000 title claims abstract description 36
- 230000006698 induction Effects 0.000 title description 5
- 102000039446 nucleic acids Human genes 0.000 claims abstract description 73
- 108020004707 nucleic acids Proteins 0.000 claims abstract description 73
- 150000007523 nucleic acids Chemical class 0.000 claims abstract description 73
- 230000014509 gene expression Effects 0.000 claims abstract description 53
- 210000000612 antigen-presenting cell Anatomy 0.000 claims abstract description 27
- 230000002519 immonomodulatory effect Effects 0.000 claims abstract description 25
- 230000005745 host immune response Effects 0.000 claims abstract description 10
- 230000001939 inductive effect Effects 0.000 claims abstract description 6
- 210000004027 cell Anatomy 0.000 claims description 70
- 108090000623 proteins and genes Proteins 0.000 claims description 70
- 210000004443 dendritic cell Anatomy 0.000 claims description 67
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 34
- 102000003814 Interleukin-10 Human genes 0.000 claims description 31
- 108090000174 Interleukin-10 Proteins 0.000 claims description 31
- 229940076144 interleukin-10 Drugs 0.000 claims description 31
- 241000701161 unidentified adenovirus Species 0.000 claims description 30
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims description 24
- 239000000427 antigen Substances 0.000 claims description 19
- 238000012217 deletion Methods 0.000 claims description 19
- 230000037430 deletion Effects 0.000 claims description 19
- 241000701022 Cytomegalovirus Species 0.000 claims description 17
- 102000036639 antigens Human genes 0.000 claims description 17
- 108091007433 antigens Proteins 0.000 claims description 17
- 101150089023 FASLG gene Proteins 0.000 claims description 14
- 102000004190 Enzymes Human genes 0.000 claims description 8
- 108090000790 Enzymes Proteins 0.000 claims description 8
- 230000003612 virological effect Effects 0.000 claims description 8
- 102100031988 Tumor necrosis factor ligand superfamily member 6 Human genes 0.000 claims description 7
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 6
- 108010076282 Factor IX Proteins 0.000 claims description 6
- 108010054218 Factor VIII Proteins 0.000 claims description 6
- 102000001690 Factor VIII Human genes 0.000 claims description 6
- 229960004222 factor ix Drugs 0.000 claims description 6
- 229960000301 factor viii Drugs 0.000 claims description 6
- 108700032588 Baculovirus p35 Proteins 0.000 claims description 5
- 102000004127 Cytokines Human genes 0.000 claims description 5
- 108090000695 Cytokines Proteins 0.000 claims description 5
- 230000002132 lysosomal effect Effects 0.000 claims description 5
- 239000002753 trypsin inhibitor Substances 0.000 claims description 5
- 108050002568 Tumor necrosis factor ligand superfamily member 6 Proteins 0.000 claims description 4
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims description 3
- 102000055025 Adenosine deaminases Human genes 0.000 claims description 3
- 108010039471 Fas Ligand Protein Proteins 0.000 claims description 3
- 108020004440 Thymidine kinase Proteins 0.000 claims description 3
- LKKMLIBUAXYLOY-UHFFFAOYSA-N 3-Amino-1-methyl-5H-pyrido[4,3-b]indole Chemical compound N1C2=CC=CC=C2C2=C1C=C(N)N=C2C LKKMLIBUAXYLOY-UHFFFAOYSA-N 0.000 claims description 2
- 102100023804 Coagulation factor VII Human genes 0.000 claims description 2
- 108010023321 Factor VII Proteins 0.000 claims description 2
- 101000578784 Homo sapiens Melanoma antigen recognized by T-cells 1 Proteins 0.000 claims description 2
- 102100031413 L-dopachrome tautomerase Human genes 0.000 claims description 2
- 101710093778 L-dopachrome tautomerase Proteins 0.000 claims description 2
- 102100022430 Melanocyte protein PMEL Human genes 0.000 claims description 2
- 102100028389 Melanoma antigen recognized by T-cells 1 Human genes 0.000 claims description 2
- 101710173694 Short transient receptor potential channel 2 Proteins 0.000 claims description 2
- 101800001271 Surface protein Proteins 0.000 claims description 2
- 102000006601 Thymidine Kinase Human genes 0.000 claims description 2
- 229940012413 factor vii Drugs 0.000 claims description 2
- 239000003102 growth factor Substances 0.000 claims description 2
- 229940088597 hormone Drugs 0.000 claims description 2
- 239000005556 hormone Substances 0.000 claims description 2
- 108010047303 von Willebrand Factor Proteins 0.000 claims description 2
- 102100036537 von Willebrand factor Human genes 0.000 claims description 2
- 229960001134 von willebrand factor Drugs 0.000 claims description 2
- 108010027410 Adenovirus E3 Proteins Proteins 0.000 claims 1
- 102000008371 intracellularly ATP-gated chloride channel activity proteins Human genes 0.000 claims 1
- 230000002085 persistent effect Effects 0.000 abstract description 10
- 210000005210 lymphoid organ Anatomy 0.000 abstract description 7
- 230000002829 reductive effect Effects 0.000 abstract description 7
- 210000001165 lymph node Anatomy 0.000 abstract description 6
- 230000028993 immune response Effects 0.000 description 27
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 description 23
- 102100030698 Interleukin-12 subunit alpha Human genes 0.000 description 23
- 229960003444 immunosuppressant agent Drugs 0.000 description 19
- 239000003018 immunosuppressive agent Substances 0.000 description 19
- 230000001861 immunosuppressant effect Effects 0.000 description 18
- 201000003883 Cystic fibrosis Diseases 0.000 description 16
- 102000004169 proteins and genes Human genes 0.000 description 16
- 230000006907 apoptotic process Effects 0.000 description 13
- 238000001415 gene therapy Methods 0.000 description 13
- 241000700605 Viruses Species 0.000 description 12
- 238000003556 assay Methods 0.000 description 11
- -1 fast Proteins 0.000 description 11
- 238000001727 in vivo Methods 0.000 description 10
- 239000002773 nucleotide Substances 0.000 description 10
- 125000003729 nucleotide group Chemical group 0.000 description 10
- 108020004414 DNA Proteins 0.000 description 9
- 241001465754 Metazoa Species 0.000 description 9
- 238000001990 intravenous administration Methods 0.000 description 9
- 239000013612 plasmid Substances 0.000 description 9
- 108091033319 polynucleotide Proteins 0.000 description 9
- 102000040430 polynucleotide Human genes 0.000 description 9
- 239000002157 polynucleotide Substances 0.000 description 9
- 238000012546 transfer Methods 0.000 description 9
- 210000004369 blood Anatomy 0.000 description 8
- 239000008280 blood Substances 0.000 description 8
- 238000010367 cloning Methods 0.000 description 8
- 210000001151 cytotoxic T lymphocyte Anatomy 0.000 description 8
- 230000006870 function Effects 0.000 description 8
- 238000000338 in vitro Methods 0.000 description 8
- 230000001404 mediated effect Effects 0.000 description 8
- 102000003951 Erythropoietin Human genes 0.000 description 7
- 108090000394 Erythropoietin Proteins 0.000 description 7
- 101150064015 FAS gene Proteins 0.000 description 7
- 241000699670 Mus sp. Species 0.000 description 7
- 230000002411 adverse Effects 0.000 description 7
- 229940105423 erythropoietin Drugs 0.000 description 7
- 208000015181 infectious disease Diseases 0.000 description 7
- 101150066555 lacZ gene Proteins 0.000 description 7
- OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 description 7
- 108091026890 Coding region Proteins 0.000 description 6
- 101100044298 Drosophila melanogaster fand gene Proteins 0.000 description 6
- 101100335198 Pneumocystis carinii fol1 gene Proteins 0.000 description 6
- 108010067390 Viral Proteins Proteins 0.000 description 6
- 230000001900 immune effect Effects 0.000 description 6
- 238000002347 injection Methods 0.000 description 6
- 239000007924 injection Substances 0.000 description 6
- 238000004806 packaging method and process Methods 0.000 description 6
- 230000002688 persistence Effects 0.000 description 6
- 230000010076 replication Effects 0.000 description 6
- 230000004044 response Effects 0.000 description 6
- 210000001519 tissue Anatomy 0.000 description 6
- 102000011045 Chloride Channels Human genes 0.000 description 5
- 108010062745 Chloride Channels Proteins 0.000 description 5
- 241000699666 Mus <mouse, genus> Species 0.000 description 5
- 206010028980 Neoplasm Diseases 0.000 description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 description 5
- 201000010099 disease Diseases 0.000 description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 5
- 210000002540 macrophage Anatomy 0.000 description 5
- 238000004519 manufacturing process Methods 0.000 description 5
- 230000037361 pathway Effects 0.000 description 5
- 210000000952 spleen Anatomy 0.000 description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 4
- 101150029409 CFTR gene Proteins 0.000 description 4
- 102000013462 Interleukin-12 Human genes 0.000 description 4
- 108010065805 Interleukin-12 Proteins 0.000 description 4
- 108700019961 Neoplasm Genes Proteins 0.000 description 4
- 102000048850 Neoplasm Genes Human genes 0.000 description 4
- 230000024932 T cell mediated immunity Effects 0.000 description 4
- 108700005077 Viral Genes Proteins 0.000 description 4
- 239000002253 acid Substances 0.000 description 4
- 230000004075 alteration Effects 0.000 description 4
- 238000010171 animal model Methods 0.000 description 4
- 210000001185 bone marrow Anatomy 0.000 description 4
- 239000003795 chemical substances by application Substances 0.000 description 4
- 229940079593 drug Drugs 0.000 description 4
- 239000003814 drug Substances 0.000 description 4
- 229940117681 interleukin-12 Drugs 0.000 description 4
- 238000007918 intramuscular administration Methods 0.000 description 4
- 239000003550 marker Substances 0.000 description 4
- MYWUZJCMWCOHBA-VIFPVBQESA-N methamphetamine Chemical compound CN[C@@H](C)CC1=CC=CC=C1 MYWUZJCMWCOHBA-VIFPVBQESA-N 0.000 description 4
- 230000003389 potentiating effect Effects 0.000 description 4
- 230000001105 regulatory effect Effects 0.000 description 4
- 230000008685 targeting Effects 0.000 description 4
- 238000013518 transcription Methods 0.000 description 4
- 230000035897 transcription Effects 0.000 description 4
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 3
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 3
- 206010011968 Decreased immune responsiveness Diseases 0.000 description 3
- 208000024720 Fabry Disease Diseases 0.000 description 3
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 description 3
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 description 3
- 102000000588 Interleukin-2 Human genes 0.000 description 3
- 108010002350 Interleukin-2 Proteins 0.000 description 3
- 108700026244 Open Reading Frames Proteins 0.000 description 3
- 102000011755 Phosphoglycerate Kinase Human genes 0.000 description 3
- 239000002202 Polyethylene glycol Substances 0.000 description 3
- 241000144282 Sigmodon Species 0.000 description 3
- 101001099217 Thermotoga maritima (strain ATCC 43589 / DSM 3109 / JCM 10099 / NBRC 100826 / MSB8) Triosephosphate isomerase Proteins 0.000 description 3
- 230000004913 activation Effects 0.000 description 3
- 210000001552 airway epithelial cell Anatomy 0.000 description 3
- 238000013459 approach Methods 0.000 description 3
- 210000003719 b-lymphocyte Anatomy 0.000 description 3
- 239000002299 complementary DNA Substances 0.000 description 3
- 230000006378 damage Effects 0.000 description 3
- 230000007547 defect Effects 0.000 description 3
- 238000001514 detection method Methods 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 230000028996 humoral immune response Effects 0.000 description 3
- 230000004727 humoral immunity Effects 0.000 description 3
- 238000003384 imaging method Methods 0.000 description 3
- 210000000987 immune system Anatomy 0.000 description 3
- 230000036039 immunity Effects 0.000 description 3
- 230000028709 inflammatory response Effects 0.000 description 3
- 230000015788 innate immune response Effects 0.000 description 3
- 238000010253 intravenous injection Methods 0.000 description 3
- 230000007246 mechanism Effects 0.000 description 3
- 108020004999 messenger RNA Proteins 0.000 description 3
- 238000010369 molecular cloning Methods 0.000 description 3
- 210000005259 peripheral blood Anatomy 0.000 description 3
- 239000011886 peripheral blood Substances 0.000 description 3
- 229920001223 polyethylene glycol Polymers 0.000 description 3
- 229920001184 polypeptide Polymers 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 230000001177 retroviral effect Effects 0.000 description 3
- 229940063675 spermine Drugs 0.000 description 3
- 230000004936 stimulating effect Effects 0.000 description 3
- 230000001052 transient effect Effects 0.000 description 3
- 230000010415 tropism Effects 0.000 description 3
- OPIFSICVWOWJMJ-AEOCFKNESA-N 5-bromo-4-chloro-3-indolyl beta-D-galactoside Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1OC1=CNC2=CC=C(Br)C(Cl)=C12 OPIFSICVWOWJMJ-AEOCFKNESA-N 0.000 description 2
- 108020005544 Antisense RNA Proteins 0.000 description 2
- 101710081722 Antitrypsin Proteins 0.000 description 2
- 108091007065 BIRCs Proteins 0.000 description 2
- 210000001266 CD8-positive T-lymphocyte Anatomy 0.000 description 2
- 241000282693 Cercopithecidae Species 0.000 description 2
- 229920002491 Diethylaminoethyl-dextran Polymers 0.000 description 2
- 238000002965 ELISA Methods 0.000 description 2
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 2
- 101710155188 Hexon-interlacing protein Proteins 0.000 description 2
- 102000016871 Hexosaminidase A Human genes 0.000 description 2
- 108010053317 Hexosaminidase A Proteins 0.000 description 2
- 101001033233 Homo sapiens Interleukin-10 Proteins 0.000 description 2
- 102000055031 Inhibitor of Apoptosis Proteins Human genes 0.000 description 2
- 102000004882 Lipase Human genes 0.000 description 2
- 108090001060 Lipase Proteins 0.000 description 2
- 208000015439 Lysosomal storage disease Diseases 0.000 description 2
- 102000018697 Membrane Proteins Human genes 0.000 description 2
- 108010052285 Membrane Proteins Proteins 0.000 description 2
- 241000713869 Moloney murine leukemia virus Species 0.000 description 2
- 241001529936 Murinae Species 0.000 description 2
- 241000288906 Primates Species 0.000 description 2
- 241000700159 Rattus Species 0.000 description 2
- 108700008625 Reporter Genes Proteins 0.000 description 2
- 241000700584 Simplexvirus Species 0.000 description 2
- 230000033540 T cell apoptotic process Effects 0.000 description 2
- 230000005867 T cell response Effects 0.000 description 2
- 102100038313 Transcription factor E2-alpha Human genes 0.000 description 2
- 239000004480 active ingredient Substances 0.000 description 2
- 239000000443 aerosol Substances 0.000 description 2
- 238000004458 analytical method Methods 0.000 description 2
- 230000001475 anti-trypsic effect Effects 0.000 description 2
- 230000005875 antibody response Effects 0.000 description 2
- 125000002091 cationic group Chemical group 0.000 description 2
- 230000001413 cellular effect Effects 0.000 description 2
- 230000007969 cellular immunity Effects 0.000 description 2
- 238000011260 co-administration Methods 0.000 description 2
- 239000003184 complementary RNA Substances 0.000 description 2
- 238000010276 construction Methods 0.000 description 2
- 230000002596 correlated effect Effects 0.000 description 2
- 230000003247 decreasing effect Effects 0.000 description 2
- 230000002950 deficient Effects 0.000 description 2
- 238000011161 development Methods 0.000 description 2
- 230000004069 differentiation Effects 0.000 description 2
- 239000012636 effector Substances 0.000 description 2
- 238000005516 engineering process Methods 0.000 description 2
- 239000003623 enhancer Substances 0.000 description 2
- 230000002255 enzymatic effect Effects 0.000 description 2
- 230000012010 growth Effects 0.000 description 2
- 238000002744 homologous recombination Methods 0.000 description 2
- 230000006801 homologous recombination Effects 0.000 description 2
- 102000052620 human IL10 Human genes 0.000 description 2
- 238000003018 immunoassay Methods 0.000 description 2
- 230000002163 immunogen Effects 0.000 description 2
- 230000009851 immunogenic response Effects 0.000 description 2
- 230000002779 inactivation Effects 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 230000005764 inhibitory process Effects 0.000 description 2
- 239000003999 initiator Substances 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
- 238000002955 isolation Methods 0.000 description 2
- 210000001821 langerhans cell Anatomy 0.000 description 2
- 150000002632 lipids Chemical class 0.000 description 2
- 210000004185 liver Anatomy 0.000 description 2
- 229910021645 metal ion Inorganic materials 0.000 description 2
- 230000005012 migration Effects 0.000 description 2
- 238000013508 migration Methods 0.000 description 2
- 239000000203 mixture Substances 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 description 2
- 230000003472 neutralizing effect Effects 0.000 description 2
- 230000005298 paramagnetic effect Effects 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- 244000052769 pathogen Species 0.000 description 2
- 230000002093 peripheral effect Effects 0.000 description 2
- 239000002243 precursor Substances 0.000 description 2
- 230000008569 process Effects 0.000 description 2
- 238000012545 processing Methods 0.000 description 2
- 238000009613 pulmonary function test Methods 0.000 description 2
- 238000003127 radioimmunoassay Methods 0.000 description 2
- 230000009467 reduction Effects 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 239000000243 solution Substances 0.000 description 2
- 239000000758 substrate Substances 0.000 description 2
- 230000005026 transcription initiation Effects 0.000 description 2
- 230000002103 transcriptional effect Effects 0.000 description 2
- 238000002054 transplantation Methods 0.000 description 2
- 241000701447 unidentified baculovirus Species 0.000 description 2
- 241001430294 unidentified retrovirus Species 0.000 description 2
- 239000013603 viral vector Substances 0.000 description 2
- SGKRLCUYIXIAHR-AKNGSSGZSA-N (4s,4ar,5s,5ar,6r,12ar)-4-(dimethylamino)-1,5,10,11,12a-pentahydroxy-6-methyl-3,12-dioxo-4a,5,5a,6-tetrahydro-4h-tetracene-2-carboxamide Chemical compound C1=CC=C2[C@H](C)[C@@H]([C@H](O)[C@@H]3[C@](C(O)=C(C(N)=O)C(=O)[C@H]3N(C)C)(O)C3=O)C3=C(O)C2=C1O SGKRLCUYIXIAHR-AKNGSSGZSA-N 0.000 description 1
- BFSVOASYOCHEOV-UHFFFAOYSA-N 2-diethylaminoethanol Chemical compound CCN(CC)CCO BFSVOASYOCHEOV-UHFFFAOYSA-N 0.000 description 1
- SPBDXSGPUHCETR-JFUDTMANSA-N 8883yp2r6d Chemical compound O1[C@@H](C)[C@H](O)[C@@H](OC)C[C@@H]1O[C@@H]1[C@@H](OC)C[C@H](O[C@@H]2C(=C/C[C@@H]3C[C@@H](C[C@@]4(O[C@@H]([C@@H](C)CC4)C(C)C)O3)OC(=O)[C@@H]3C=C(C)[C@@H](O)[C@H]4OC\C([C@@]34O)=C/C=C/[C@@H]2C)/C)O[C@H]1C.C1C[C@H](C)[C@@H]([C@@H](C)CC)O[C@@]21O[C@H](C\C=C(C)\[C@@H](O[C@@H]1O[C@@H](C)[C@H](O[C@@H]3O[C@@H](C)[C@H](O)[C@@H](OC)C3)[C@@H](OC)C1)[C@@H](C)\C=C\C=C/1[C@]3([C@H](C(=O)O4)C=C(C)[C@@H](O)[C@H]3OC\1)O)C[C@H]4C2 SPBDXSGPUHCETR-JFUDTMANSA-N 0.000 description 1
- 102000006772 Acid Ceramidase Human genes 0.000 description 1
- 108020005296 Acid Ceramidase Proteins 0.000 description 1
- 241000701242 Adenoviridae Species 0.000 description 1
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 description 1
- 229940088872 Apoptosis inhibitor Drugs 0.000 description 1
- 108020000946 Bacterial DNA Proteins 0.000 description 1
- 108010039209 Blood Coagulation Factors Proteins 0.000 description 1
- 102000015081 Blood Coagulation Factors Human genes 0.000 description 1
- 101710132601 Capsid protein Proteins 0.000 description 1
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 108091006146 Channels Proteins 0.000 description 1
- 208000017667 Chronic Disease Diseases 0.000 description 1
- 206010053567 Coagulopathies Diseases 0.000 description 1
- 101710094648 Coat protein Proteins 0.000 description 1
- 208000003322 Coinfection Diseases 0.000 description 1
- 241000557626 Corvus corax Species 0.000 description 1
- 108010051219 Cre recombinase Proteins 0.000 description 1
- 102000000311 Cytosine Deaminase Human genes 0.000 description 1
- 108010080611 Cytosine Deaminase Proteins 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 230000007067 DNA methylation Effects 0.000 description 1
- 230000004543 DNA replication Effects 0.000 description 1
- 206010011878 Deafness Diseases 0.000 description 1
- 229920002307 Dextran Polymers 0.000 description 1
- 102000001039 Dystrophin Human genes 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- UPEZCKBFRMILAV-JNEQICEOSA-N Ecdysone Natural products O=C1[C@H]2[C@@](C)([C@@H]3C([C@@]4(O)[C@@](C)([C@H]([C@H]([C@@H](O)CCC(O)(C)C)C)CC4)CC3)=C1)C[C@H](O)[C@H](O)C2 UPEZCKBFRMILAV-JNEQICEOSA-N 0.000 description 1
- 241000588724 Escherichia coli Species 0.000 description 1
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 description 1
- 208000033149 Farber disease Diseases 0.000 description 1
- 201000008892 GM1 Gangliosidosis Diseases 0.000 description 1
- 102100028496 Galactocerebrosidase Human genes 0.000 description 1
- 208000017462 Galactosialidosis Diseases 0.000 description 1
- 108010042681 Galactosylceramidase Proteins 0.000 description 1
- 208000015872 Gaucher disease Diseases 0.000 description 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 description 1
- 102000004547 Glucosylceramidase Human genes 0.000 description 1
- 108010017544 Glucosylceramidase Proteins 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 1
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 description 1
- 108010017213 Granulocyte-Macrophage Colony-Stimulating Factor Proteins 0.000 description 1
- 102100039620 Granulocyte-macrophage colony-stimulating factor Human genes 0.000 description 1
- 208000028782 Hereditary disease Diseases 0.000 description 1
- 102000016870 Hexosaminidase B Human genes 0.000 description 1
- 108010053345 Hexosaminidase B Proteins 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000718525 Homo sapiens Alpha-galactosidase A Proteins 0.000 description 1
- 101000765010 Homo sapiens Beta-galactosidase Proteins 0.000 description 1
- 101000907783 Homo sapiens Cystic fibrosis transmembrane conductance regulator Proteins 0.000 description 1
- 241000701044 Human gammaherpesvirus 4 Species 0.000 description 1
- 208000015178 Hurler syndrome Diseases 0.000 description 1
- 108010053927 Iduronate Sulfatase Proteins 0.000 description 1
- 108700002232 Immediate-Early Genes Proteins 0.000 description 1
- 108060003951 Immunoglobulin Proteins 0.000 description 1
- 102100021592 Interleukin-7 Human genes 0.000 description 1
- 108010002586 Interleukin-7 Proteins 0.000 description 1
- 208000028226 Krabbe disease Diseases 0.000 description 1
- 231100000416 LDH assay Toxicity 0.000 description 1
- 102000003960 Ligases Human genes 0.000 description 1
- 108090000364 Ligases Proteins 0.000 description 1
- 239000004367 Lipase Substances 0.000 description 1
- 239000004472 Lysine Substances 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- 208000033868 Lysosomal disease Diseases 0.000 description 1
- 102000043129 MHC class I family Human genes 0.000 description 1
- 108091054437 MHC class I family Proteins 0.000 description 1
- 102000043131 MHC class II family Human genes 0.000 description 1
- 108091054438 MHC class II family Proteins 0.000 description 1
- 101710125418 Major capsid protein Proteins 0.000 description 1
- 208000024556 Mendelian disease Diseases 0.000 description 1
- 241000713333 Mouse mammary tumor virus Species 0.000 description 1
- 208000008955 Mucolipidoses Diseases 0.000 description 1
- 206010072928 Mucolipidosis type II Diseases 0.000 description 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 description 1
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 description 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 description 1
- 241000699660 Mus musculus Species 0.000 description 1
- 101000638240 Mus musculus Tumor necrosis factor ligand superfamily member 6 Proteins 0.000 description 1
- 208000034827 Neointima Diseases 0.000 description 1
- 229930193140 Neomycin Natural products 0.000 description 1
- 102000005348 Neuraminidase Human genes 0.000 description 1
- 108010006232 Neuraminidase Proteins 0.000 description 1
- 208000014060 Niemann-Pick disease Diseases 0.000 description 1
- 238000000636 Northern blotting Methods 0.000 description 1
- 101710141454 Nucleoprotein Proteins 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 description 1
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 101710083689 Probable capsid protein Proteins 0.000 description 1
- 108010076504 Protein Sorting Signals Proteins 0.000 description 1
- 108020004511 Recombinant DNA Proteins 0.000 description 1
- 241001068263 Replication competent viruses Species 0.000 description 1
- 241000714474 Rous sarcoma virus Species 0.000 description 1
- 208000021811 Sandhoff disease Diseases 0.000 description 1
- 238000002105 Southern blotting Methods 0.000 description 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 description 1
- 230000006044 T cell activation Effects 0.000 description 1
- 230000020385 T cell costimulation Effects 0.000 description 1
- 208000022292 Tay-Sachs disease Diseases 0.000 description 1
- 108020005038 Terminator Codon Proteins 0.000 description 1
- 239000004098 Tetracycline Substances 0.000 description 1
- 241000906446 Theraps Species 0.000 description 1
- 102000036693 Thrombopoietin Human genes 0.000 description 1
- 108010041111 Thrombopoietin Proteins 0.000 description 1
- 206010052779 Transplant rejections Diseases 0.000 description 1
- 108700001567 Type I Schindler Disease Proteins 0.000 description 1
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 description 1
- 108091008605 VEGF receptors Proteins 0.000 description 1
- 102000009484 Vascular Endothelial Growth Factor Receptors Human genes 0.000 description 1
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 description 1
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 description 1
- 108020005202 Viral DNA Proteins 0.000 description 1
- 108010087302 Viral Structural Proteins Proteins 0.000 description 1
- 208000036142 Viral infection Diseases 0.000 description 1
- 208000026589 Wolman disease Diseases 0.000 description 1
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 230000006786 activation induced cell death Effects 0.000 description 1
- 239000012190 activator Substances 0.000 description 1
- 108700010877 adenoviridae proteins Proteins 0.000 description 1
- 230000006154 adenylylation Effects 0.000 description 1
- 238000007818 agglutination assay Methods 0.000 description 1
- 230000000961 alloantigen Effects 0.000 description 1
- 230000000735 allogeneic effect Effects 0.000 description 1
- UPEZCKBFRMILAV-UHFFFAOYSA-N alpha-Ecdysone Natural products C1C(O)C(O)CC2(C)C(CCC3(C(C(C(O)CCC(C)(C)O)C)CCC33O)C)C3=CC(=O)C21 UPEZCKBFRMILAV-UHFFFAOYSA-N 0.000 description 1
- 102000016679 alpha-Glucosidases Human genes 0.000 description 1
- 108010028144 alpha-Glucosidases Proteins 0.000 description 1
- 230000000840 anti-viral effect Effects 0.000 description 1
- 239000000158 apoptosis inhibitor Substances 0.000 description 1
- 230000005775 apoptotic pathway Effects 0.000 description 1
- 208000025341 autosomal recessive disease Diseases 0.000 description 1
- 230000008901 benefit Effects 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000003114 blood coagulation factor Substances 0.000 description 1
- 210000001715 carotid artery Anatomy 0.000 description 1
- 230000010261 cell growth Effects 0.000 description 1
- 230000022534 cell killing Effects 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 230000006037 cell lysis Effects 0.000 description 1
- 230000010001 cellular homeostasis Effects 0.000 description 1
- 238000005119 centrifugation Methods 0.000 description 1
- 238000012512 characterization method Methods 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 238000012411 cloning technique Methods 0.000 description 1
- 230000035602 clotting Effects 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 230000004154 complement system Effects 0.000 description 1
- 238000013329 compounding Methods 0.000 description 1
- 238000007796 conventional method Methods 0.000 description 1
- 230000000875 corresponding effect Effects 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 230000016396 cytokine production Effects 0.000 description 1
- 230000008260 defense mechanism Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- 230000003467 diminishing effect Effects 0.000 description 1
- 231100000673 dose–response relationship Toxicity 0.000 description 1
- 230000003828 downregulation Effects 0.000 description 1
- 229960003722 doxycycline Drugs 0.000 description 1
- UPEZCKBFRMILAV-JMZLNJERSA-N ecdysone Chemical compound C1[C@@H](O)[C@@H](O)C[C@]2(C)[C@@H](CC[C@@]3([C@@H]([C@@H]([C@H](O)CCC(C)(C)O)C)CC[C@]33O)C)C3=CC(=O)[C@@H]21 UPEZCKBFRMILAV-JMZLNJERSA-N 0.000 description 1
- 230000008030 elimination Effects 0.000 description 1
- 238000003379 elimination reaction Methods 0.000 description 1
- 230000010502 episomal replication Effects 0.000 description 1
- 210000002919 epithelial cell Anatomy 0.000 description 1
- 210000000981 epithelium Anatomy 0.000 description 1
- 239000013613 expression plasmid Substances 0.000 description 1
- 108010052621 fas Receptor Proteins 0.000 description 1
- 102000018823 fas Receptor Human genes 0.000 description 1
- 210000002950 fibroblast Anatomy 0.000 description 1
- 238000000684 flow cytometry Methods 0.000 description 1
- 238000009472 formulation Methods 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 238000002825 functional assay Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 238000010362 genome editing Methods 0.000 description 1
- 201000004502 glycogen storage disease II Diseases 0.000 description 1
- 239000001963 growth medium Substances 0.000 description 1
- 108010089932 heparan sulfate sulfatase Proteins 0.000 description 1
- KLHSDMQFUVANEB-MELZOAELSA-L hexadecyl-[(2r,3r)-4-[hexadecyl(dimethyl)azaniumyl]-2,3-dimethoxybutyl]-dimethylazanium;dibromide Chemical compound [Br-].[Br-].CCCCCCCCCCCCCCCC[N+](C)(C)C[C@@H](OC)[C@H](OC)C[N+](C)(C)CCCCCCCCCCCCCCCC KLHSDMQFUVANEB-MELZOAELSA-L 0.000 description 1
- 230000001744 histochemical effect Effects 0.000 description 1
- 102000043404 human GLA Human genes 0.000 description 1
- 239000012216 imaging agent Substances 0.000 description 1
- 230000005847 immunogenicity Effects 0.000 description 1
- 102000018358 immunoglobulin Human genes 0.000 description 1
- 238000011532 immunohistochemical staining Methods 0.000 description 1
- 230000006054 immunological memory Effects 0.000 description 1
- 238000001114 immunoprecipitation Methods 0.000 description 1
- 230000003308 immunostimulating effect Effects 0.000 description 1
- 230000001506 immunosuppresive effect Effects 0.000 description 1
- 230000006872 improvement Effects 0.000 description 1
- 238000011503 in vivo imaging Methods 0.000 description 1
- 239000012678 infectious agent Substances 0.000 description 1
- 230000002458 infectious effect Effects 0.000 description 1
- 230000002757 inflammatory effect Effects 0.000 description 1
- 238000002743 insertional mutagenesis Methods 0.000 description 1
- 230000010354 integration Effects 0.000 description 1
- 229940100994 interleukin-7 Drugs 0.000 description 1
- 210000003292 kidney cell Anatomy 0.000 description 1
- 230000002147 killing effect Effects 0.000 description 1
- 238000011813 knockout mouse model Methods 0.000 description 1
- 238000002843 lactate dehydrogenase assay Methods 0.000 description 1
- 239000003446 ligand Substances 0.000 description 1
- 235000019421 lipase Nutrition 0.000 description 1
- 239000007788 liquid Substances 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 230000005980 lung dysfunction Effects 0.000 description 1
- 210000002751 lymph Anatomy 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 229920002521 macromolecule Polymers 0.000 description 1
- 238000002595 magnetic resonance imaging Methods 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 230000035800 maturation Effects 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 230000002503 metabolic effect Effects 0.000 description 1
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 description 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 description 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 210000000822 natural killer cell Anatomy 0.000 description 1
- 230000008692 neointimal formation Effects 0.000 description 1
- 229960004927 neomycin Drugs 0.000 description 1
- 230000001613 neoplastic effect Effects 0.000 description 1
- 238000011580 nude mouse model Methods 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 244000045947 parasite Species 0.000 description 1
- 230000001717 pathogenic effect Effects 0.000 description 1
- 238000005375 photometry Methods 0.000 description 1
- 101150088856 pix gene Proteins 0.000 description 1
- 229920001515 polyalkylene glycol Polymers 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 238000002600 positron emission tomography Methods 0.000 description 1
- 239000000843 powder Substances 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 230000002265 prevention Effects 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 230000000644 propagated effect Effects 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 230000009325 pulmonary function Effects 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
- 108010043277 recombinant soluble CD4 Proteins 0.000 description 1
- 210000001533 respiratory mucosa Anatomy 0.000 description 1
- 210000002345 respiratory system Anatomy 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000003757 reverse transcription PCR Methods 0.000 description 1
- 230000002441 reversible effect Effects 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
- 238000000926 separation method Methods 0.000 description 1
- 238000002603 single-photon emission computed tomography Methods 0.000 description 1
- 238000013125 spirometry Methods 0.000 description 1
- 230000003393 splenic effect Effects 0.000 description 1
- 230000006641 stabilisation Effects 0.000 description 1
- 238000011105 stabilization Methods 0.000 description 1
- 210000000130 stem cell Anatomy 0.000 description 1
- 230000000638 stimulation Effects 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 230000002123 temporal effect Effects 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 210000001550 testis Anatomy 0.000 description 1
- 229960002180 tetracycline Drugs 0.000 description 1
- 229930101283 tetracycline Natural products 0.000 description 1
- 235000019364 tetracycline Nutrition 0.000 description 1
- 150000003522 tetracyclines Chemical class 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 230000003614 tolerogenic effect Effects 0.000 description 1
- 238000011200 topical administration Methods 0.000 description 1
- 230000005030 transcription termination Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 230000001131 transforming effect Effects 0.000 description 1
- 230000014616 translation Effects 0.000 description 1
- 210000004881 tumor cell Anatomy 0.000 description 1
- 230000003827 upregulation Effects 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
- 210000003556 vascular endothelial cell Anatomy 0.000 description 1
- 230000006648 viral gene expression Effects 0.000 description 1
- 230000029812 viral genome replication Effects 0.000 description 1
- 230000009385 viral infection Effects 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 238000012800 visualization Methods 0.000 description 1
- 238000001262 western blot Methods 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
- A61K39/4615—Dendritic cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/462—Cellular immunotherapy characterized by the effect or the function of the cells
- A61K39/4622—Antigen presenting cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/46434—Antigens related to induction of tolerance to non-self
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464402—Receptors, cell surface antigens or cell surface determinants
- A61K39/464416—Receptors for cytokines
- A61K39/464417—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/46443—Growth factors
- A61K39/464434—Transforming growth factor [TGF]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464436—Cytokines
- A61K39/46444—Interleukins [IL]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
- C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Cell Biology (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Oncology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Molecular Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US13627899P | 1999-05-27 | 1999-05-27 | |
US60/136,278 | 1999-05-27 | ||
PCT/US2000/014344 WO2000073477A1 (en) | 1999-05-27 | 2000-05-25 | Methods for induction of tolerance to adenoviral vectors and transgene products |
Publications (1)
Publication Number | Publication Date |
---|---|
CA2373547A1 true CA2373547A1 (en) | 2000-12-07 |
Family
ID=22472151
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA002373547A Abandoned CA2373547A1 (en) | 1999-05-27 | 2000-05-25 | Methods for induction of tolerance to adenoviral vectors and transgene products |
Country Status (5)
Country | Link |
---|---|
EP (1) | EP1180156A1 (de) |
JP (1) | JP2003501040A (de) |
AU (1) | AU5161600A (de) |
CA (1) | CA2373547A1 (de) |
WO (1) | WO2000073477A1 (de) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU782255B2 (en) * | 1999-10-12 | 2005-07-14 | Lophius Biosciences, GmbH | Gene transfer vectors for treating autoimmune diseases and diseases with immunopathogenesis by therapy |
AU2001234508A1 (en) * | 2000-02-02 | 2001-08-14 | Genzyme Corporation | Methods for treatment of restenosis using adenoviral vectors and transgene products |
JP4237449B2 (ja) * | 2002-06-05 | 2009-03-11 | 国立医薬品食品衛生研究所長 | アデノウィルスベクター |
DE10254374A1 (de) * | 2002-11-21 | 2004-06-03 | Universitätsklinikum Hamburg-Eppendorf Körperschaft des Öffentlichen Rechts | Adenovirale Vektoren zum Transfer von spezifischen Genen in Körperzellen |
Family Cites Families (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1998052615A1 (en) * | 1997-05-22 | 1998-11-26 | Uab Research Foundation | Controlling immune response to specific antigens |
-
2000
- 2000-05-25 EP EP00936276A patent/EP1180156A1/de not_active Withdrawn
- 2000-05-25 WO PCT/US2000/014344 patent/WO2000073477A1/en active Search and Examination
- 2000-05-25 CA CA002373547A patent/CA2373547A1/en not_active Abandoned
- 2000-05-25 JP JP2001500789A patent/JP2003501040A/ja active Pending
- 2000-05-25 AU AU51616/00A patent/AU5161600A/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
WO2000073477A1 (en) | 2000-12-07 |
EP1180156A1 (de) | 2002-02-20 |
JP2003501040A (ja) | 2003-01-14 |
AU5161600A (en) | 2000-12-18 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
AU727992B2 (en) | Adenoviral vectors comprising a modified E4 region but retaining E4ORF3 | |
EP0787200B1 (de) | Rekombinanter adenovirus und methoden zu dessen verwendung | |
Becker et al. | Use of recombinant adenovirus for metabolic engineering of mammalian cells | |
Christ et al. | Gene therapy with recombinant adenovirus vectors: evaluation of the host immune response | |
JP3868489B2 (ja) | リポタンパク質の代謝における欠陥の治療のための遺伝子治療の方法及び組成物 | |
US20030092162A1 (en) | Chimeric adenoviral vectors for targeted gene delivery | |
CA2287472A1 (en) | Novel adenoviral vectors capable of facilitating increased persistence of transgene expression | |
CA2287076A1 (en) | Novel transgene expression system for increased persistence | |
JP2000106875A (ja) | 目的遺伝子の発現を改善するためのアデノウイルスe4リ―ディングフレ―ムの使用 | |
WO1999057296A1 (en) | Partially deleted adenoviral vectors | |
US20040023389A1 (en) | Adenoviral vectors having nucleic acids encoding immunomodulatory molecules | |
Lieber et al. | Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo | |
KR20030072214A (ko) | 완화된 부작용을 갖는 신규 재조합 아데노바이러스 벡터 | |
JP2002512785A (ja) | 疾患治療用のアデノウイルスベクター | |
CA2373547A1 (en) | Methods for induction of tolerance to adenoviral vectors and transgene products | |
JP2003504316A (ja) | 疾患を処置するためのアデノウイルスベクター | |
JP2002508974A (ja) | 持続性遺伝子発現のための新規なプロモーターエレメント | |
CA2328087A1 (en) | Partially deleted adenoviral vectors | |
WO2001057228A1 (en) | Methods for treatment of restenosis using adenoviral vectors and transgene products | |
AU3142902A (en) | Novel transgene expression system for increased persistence |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
FZDE | Discontinued |