JP7258031B2 - 機能性核酸分子及びその使用 - Google Patents
機能性核酸分子及びその使用 Download PDFInfo
- Publication number
- JP7258031B2 JP7258031B2 JP2020537887A JP2020537887A JP7258031B2 JP 7258031 B2 JP7258031 B2 JP 7258031B2 JP 2020537887 A JP2020537887 A JP 2020537887A JP 2020537887 A JP2020537887 A JP 2020537887A JP 7258031 B2 JP7258031 B2 JP 7258031B2
- Authority
- JP
- Japan
- Prior art keywords
- ires
- nucleic acid
- sequence
- functional nucleic
- acid molecule
- 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.)
- Active
Links
- 150000007523 nucleic acids Chemical class 0.000 title claims description 238
- 102000039446 nucleic acids Human genes 0.000 title claims description 234
- 108020004707 nucleic acids Proteins 0.000 title claims description 234
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 claims description 417
- 108020004999 messenger RNA Proteins 0.000 claims description 118
- 230000002441 reversible effect Effects 0.000 claims description 77
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 66
- 239000002773 nucleotide Substances 0.000 claims description 61
- 125000003729 nucleotide group Chemical group 0.000 claims description 60
- 108090000623 proteins and genes Proteins 0.000 claims description 54
- 230000014509 gene expression Effects 0.000 claims description 50
- 230000027455 binding Effects 0.000 claims description 45
- 230000014616 translation Effects 0.000 claims description 43
- 238000013519 translation Methods 0.000 claims description 28
- 230000000295 complement effect Effects 0.000 claims description 22
- 108091026890 Coding region Proteins 0.000 claims description 21
- 108020004414 DNA Proteins 0.000 claims description 20
- 230000002708 enhancing effect Effects 0.000 claims description 19
- 102000053602 DNA Human genes 0.000 claims description 17
- 239000013604 expression vector Substances 0.000 claims description 16
- 108020003589 5' Untranslated Regions Proteins 0.000 claims description 14
- 230000001105 regulatory effect Effects 0.000 claims description 13
- 239000013598 vector Substances 0.000 claims description 12
- 238000000034 method Methods 0.000 claims description 11
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims description 10
- 201000010099 disease Diseases 0.000 claims description 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 7
- 230000002829 reductive effect Effects 0.000 claims description 5
- 230000001627 detrimental effect Effects 0.000 claims description 4
- 238000000338 in vitro Methods 0.000 claims description 4
- 230000003828 downregulation Effects 0.000 claims description 3
- 125000006850 spacer group Chemical group 0.000 claims description 3
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 2
- 208000024556 Mendelian disease Diseases 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 description 130
- 241000711549 Hepacivirus C Species 0.000 description 107
- 101100239628 Danio rerio myca gene Proteins 0.000 description 59
- 239000013612 plasmid Substances 0.000 description 59
- 238000001262 western blot Methods 0.000 description 55
- 108010032428 Protein Deglycase DJ-1 Proteins 0.000 description 54
- 102000007659 Protein Deglycase DJ-1 Human genes 0.000 description 44
- 239000012636 effector Substances 0.000 description 34
- 102000007469 Actins Human genes 0.000 description 33
- 108010085238 Actins Proteins 0.000 description 33
- 238000011529 RT qPCR Methods 0.000 description 33
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 description 31
- 208000000474 Poliomyelitis Diseases 0.000 description 31
- 102000004169 proteins and genes Human genes 0.000 description 28
- 102100024108 Dystrophin Human genes 0.000 description 26
- 241000710127 Cricket paralysis virus Species 0.000 description 25
- 230000003612 virological effect Effects 0.000 description 24
- 238000001890 transfection Methods 0.000 description 21
- 241000710188 Encephalomyocarditis virus Species 0.000 description 20
- 241000991587 Enterovirus C Species 0.000 description 19
- 230000001413 cellular effect Effects 0.000 description 17
- 230000000694 effects Effects 0.000 description 17
- 238000012360 testing method Methods 0.000 description 17
- 230000006698 induction Effects 0.000 description 16
- 230000035772 mutation Effects 0.000 description 15
- 230000036515 potency Effects 0.000 description 15
- 238000012986 modification Methods 0.000 description 13
- 230000004048 modification Effects 0.000 description 13
- 239000006166 lysate Substances 0.000 description 12
- 230000008685 targeting Effects 0.000 description 12
- 230000000692 anti-sense effect Effects 0.000 description 11
- 108020004463 18S ribosomal RNA Proteins 0.000 description 10
- 230000003827 upregulation Effects 0.000 description 10
- 101001030211 Homo sapiens Myc proto-oncogene protein Proteins 0.000 description 8
- 241000700605 Viruses Species 0.000 description 7
- 239000013613 expression plasmid Substances 0.000 description 6
- 102100031702 Endoplasmic reticulum membrane sensor NFE2L1 Human genes 0.000 description 5
- 101000588298 Homo sapiens Endoplasmic reticulum membrane sensor NFE2L1 Proteins 0.000 description 5
- 101000577547 Homo sapiens Nuclear respiratory factor 1 Proteins 0.000 description 5
- 101000601727 Homo sapiens Parkinson disease protein 7 Proteins 0.000 description 5
- 101000994434 Homo sapiens Protein jagged-2 Proteins 0.000 description 5
- 101000772888 Homo sapiens Ubiquitin-protein ligase E3A Proteins 0.000 description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 description 5
- 102100032733 Protein jagged-2 Human genes 0.000 description 5
- 102100030434 Ubiquitin-protein ligase E3A Human genes 0.000 description 5
- 239000013592 cell lysate Substances 0.000 description 5
- 102000054831 human PARK7 Human genes 0.000 description 5
- 210000004962 mammalian cell Anatomy 0.000 description 5
- 108020004705 Codon Proteins 0.000 description 4
- 102100023115 Dual specificity tyrosine-phosphorylation-regulated kinase 2 Human genes 0.000 description 4
- 101001049990 Homo sapiens Dual specificity tyrosine-phosphorylation-regulated kinase 2 Proteins 0.000 description 4
- 101001064282 Homo sapiens Platelet-activating factor acetylhydrolase IB subunit beta Proteins 0.000 description 4
- 102100038895 Myc proto-oncogene protein Human genes 0.000 description 4
- 102100030655 Platelet-activating factor acetylhydrolase IB subunit beta Human genes 0.000 description 4
- 102000002278 Ribosomal Proteins Human genes 0.000 description 4
- 108010000605 Ribosomal Proteins Proteins 0.000 description 4
- 230000008901 benefit Effects 0.000 description 4
- 238000002474 experimental method Methods 0.000 description 4
- BCOSEZGCLGPUSL-UHFFFAOYSA-N 2,3,3-trichloroprop-2-enoyl chloride Chemical compound ClC(Cl)=C(Cl)C(Cl)=O BCOSEZGCLGPUSL-UHFFFAOYSA-N 0.000 description 3
- 230000004568 DNA-binding Effects 0.000 description 3
- 101000924629 Homo sapiens Apoptotic protease-activating factor 1 Proteins 0.000 description 3
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 description 3
- 108091046869 Telomeric non-coding RNA Proteins 0.000 description 3
- 230000001419 dependent effect Effects 0.000 description 3
- 230000009977 dual effect Effects 0.000 description 3
- 239000003623 enhancer Substances 0.000 description 3
- 102000050495 human APAF1 Human genes 0.000 description 3
- 229930182817 methionine Natural products 0.000 description 3
- 238000001243 protein synthesis Methods 0.000 description 3
- YIMATHOGWXZHFX-WCTZXXKLSA-N (2r,3r,4r,5r)-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolane-2,4-diol Chemical compound COCCO[C@H]1[C@H](O)O[C@H](CO)[C@H]1O YIMATHOGWXZHFX-WCTZXXKLSA-N 0.000 description 2
- GRJDAVSXJPWJNJ-RCYBNZJXSA-N 1-[(2r,3r,4s,5s)-5-[bromo(hydroxy)methyl]-3,4-dihydroxyoxolan-2-yl]pyrimidine-2,4-dione Chemical compound O[C@@H]1[C@H](O)[C@@H](C(Br)O)O[C@H]1N1C(=O)NC(=O)C=C1 GRJDAVSXJPWJNJ-RCYBNZJXSA-N 0.000 description 2
- KPPPLADORXGUFI-KCRXGDJASA-N 4-amino-1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(1-hydroxyethyl)oxolan-2-yl]pyrimidin-2-one Chemical compound O[C@@H]1[C@H](O)[C@@H](C(O)C)O[C@H]1N1C(=O)N=C(N)C=C1 KPPPLADORXGUFI-KCRXGDJASA-N 0.000 description 2
- 108020005544 Antisense RNA Proteins 0.000 description 2
- 108091028690 C-myc mRNA Proteins 0.000 description 2
- 241000206602 Eukaryota Species 0.000 description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 2
- 208000031448 Genomic Instability Diseases 0.000 description 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 description 2
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 description 2
- 206010029260 Neuroblastoma Diseases 0.000 description 2
- 102000035195 Peptidases Human genes 0.000 description 2
- 108091005804 Peptidases Proteins 0.000 description 2
- 102000005877 Peptide Initiation Factors Human genes 0.000 description 2
- 108010044843 Peptide Initiation Factors Proteins 0.000 description 2
- 229930185560 Pseudouridine Natural products 0.000 description 2
- PTJWIQPHWPFNBW-UHFFFAOYSA-N Pseudouridine C Natural products OC1C(O)C(CO)OC1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-UHFFFAOYSA-N 0.000 description 2
- 102000009572 RNA Polymerase II Human genes 0.000 description 2
- 108010009460 RNA Polymerase II Proteins 0.000 description 2
- 102000018779 Replication Protein C Human genes 0.000 description 2
- 108010027647 Replication Protein C Proteins 0.000 description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 description 2
- 108091036066 Three prime untranslated region Proteins 0.000 description 2
- 230000004913 activation Effects 0.000 description 2
- 230000006907 apoptotic process Effects 0.000 description 2
- WGDUUQDYDIIBKT-UHFFFAOYSA-N beta-Pseudouridine Natural products OC1OC(CN2C=CC(=O)NC2=O)C(O)C1O WGDUUQDYDIIBKT-UHFFFAOYSA-N 0.000 description 2
- 238000007622 bioinformatic analysis Methods 0.000 description 2
- 238000007385 chemical modification Methods 0.000 description 2
- 238000010367 cloning Methods 0.000 description 2
- 230000004186 co-expression Effects 0.000 description 2
- 239000003184 complementary RNA Substances 0.000 description 2
- 230000009918 complex formation Effects 0.000 description 2
- 238000012937 correction Methods 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 210000004265 eukaryotic small ribosome subunit Anatomy 0.000 description 2
- 206010073071 hepatocellular carcinoma Diseases 0.000 description 2
- 231100000844 hepatocellular carcinoma Toxicity 0.000 description 2
- 210000003494 hepatocyte Anatomy 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 230000002458 infectious effect Effects 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 230000003993 interaction Effects 0.000 description 2
- 210000003734 kidney Anatomy 0.000 description 2
- 239000000463 material Substances 0.000 description 2
- 230000007246 mechanism Effects 0.000 description 2
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 2
- 230000003389 potentiating effect Effects 0.000 description 2
- 235000019833 protease Nutrition 0.000 description 2
- PTJWIQPHWPFNBW-GBNDHIKLSA-N pseudouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-GBNDHIKLSA-N 0.000 description 2
- 230000009467 reduction Effects 0.000 description 2
- 230000014621 translational initiation Effects 0.000 description 2
- 108020005345 3' Untranslated Regions Proteins 0.000 description 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- 101150091518 APAF1 gene Proteins 0.000 description 1
- 108700028369 Alleles Proteins 0.000 description 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 description 1
- 206010003591 Ataxia Diseases 0.000 description 1
- 108010077544 Chromatin Proteins 0.000 description 1
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 108090000331 Firefly luciferases Proteins 0.000 description 1
- 208000028782 Hereditary disease Diseases 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 108020005198 Long Noncoding RNA Proteins 0.000 description 1
- 108060001084 Luciferase Proteins 0.000 description 1
- 241001465754 Metazoa Species 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 206010033799 Paralysis Diseases 0.000 description 1
- 241000709664 Picornaviridae Species 0.000 description 1
- 102000014450 RNA Polymerase III Human genes 0.000 description 1
- 108010078067 RNA Polymerase III Proteins 0.000 description 1
- 208000035977 Rare disease Diseases 0.000 description 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 1
- 108010052090 Renilla Luciferases Proteins 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- 108091023040 Transcription factor Proteins 0.000 description 1
- 102000040945 Transcription factor Human genes 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 230000032683 aging Effects 0.000 description 1
- 150000001413 amino acids Chemical class 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 210000003483 chromatin Anatomy 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 238000012790 confirmation Methods 0.000 description 1
- 230000007423 decrease Effects 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 210000001808 exosome Anatomy 0.000 description 1
- 230000005714 functional activity Effects 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 238000003306 harvesting Methods 0.000 description 1
- 238000013537 high throughput screening Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 230000007257 malfunction Effects 0.000 description 1
- 230000001394 metastastic effect Effects 0.000 description 1
- 206010061289 metastatic neoplasm Diseases 0.000 description 1
- 108091070501 miRNA Proteins 0.000 description 1
- 239000002679 microRNA Substances 0.000 description 1
- 238000002703 mutagenesis Methods 0.000 description 1
- 231100000350 mutagenesis Toxicity 0.000 description 1
- 239000002105 nanoparticle Substances 0.000 description 1
- 238000010899 nucleation Methods 0.000 description 1
- 230000036961 partial effect Effects 0.000 description 1
- 239000002245 particle Substances 0.000 description 1
- 230000001124 posttranscriptional effect Effects 0.000 description 1
- 238000007634 remodeling Methods 0.000 description 1
- 230000003938 response to stress Effects 0.000 description 1
- 108020004418 ribosomal RNA Proteins 0.000 description 1
- 230000001568 sexual effect Effects 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 230000035882 stress Effects 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 230000000946 synaptic effect Effects 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 230000018412 transposition, RNA-mediated Effects 0.000 description 1
- 239000013603 viral vector Substances 0.000 description 1
Images
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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- C12N15/67—General methods for enhancing the expression
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
-
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| IT102017000105372 | 2017-09-20 | ||
| IT102017000105372A IT201700105372A1 (it) | 2017-09-20 | 2017-09-20 | Molecola di acido nucleico funzionale e relativo uso |
| PCT/IB2018/057262 WO2019058304A1 (en) | 2017-09-20 | 2018-09-20 | FUNCTIONAL NUCLEIC ACID MOLECULE AND ASSOCIATED APPLICATIONS |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020535845A JP2020535845A (ja) | 2020-12-10 |
| JP2020535845A5 JP2020535845A5 (enExample) | 2021-09-09 |
| JP7258031B2 true JP7258031B2 (ja) | 2023-04-14 |
Family
ID=61024873
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020537887A Active JP7258031B2 (ja) | 2017-09-20 | 2018-09-20 | 機能性核酸分子及びその使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US11649456B2 (enExample) |
| EP (1) | EP3684928A1 (enExample) |
| JP (1) | JP7258031B2 (enExample) |
| KR (1) | KR102677300B1 (enExample) |
| CN (1) | CN111386344B (enExample) |
| CA (1) | CA3076163A1 (enExample) |
| IT (1) | IT201700105372A1 (enExample) |
| WO (1) | WO2019058304A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IT201900011490A1 (it) | 2019-07-11 | 2021-01-11 | Scuola Int Superiore Di Studi Avanzati Sissa | MOLECOLE DI ACIDO NUCLEICO FUNZIONALI CHE AUMENTANO LA TRADUZIONE DI UN mRNA DELLA FRATASSINA |
| CN110484563B (zh) * | 2019-07-25 | 2023-04-07 | 新乡医学院 | 哺乳动物细胞组合表达载体、表达系统、制备方法和应用 |
| KR20230128446A (ko) * | 2020-09-24 | 2023-09-05 | 폰다치오네 이스티튜토 이탈리아노 디 테크놀로지아 | 변형된 기능적 핵산 분자 |
| EP4225916A1 (en) | 2020-10-08 | 2023-08-16 | Transine Therapeutics Limited | Functional nucleic acid molecules |
| EP3992289A1 (en) | 2020-10-30 | 2022-05-04 | Transine Therapeutics Limited | Functional nucleic acid molecules incorporating protein binding domain |
| CN112126644B (zh) * | 2020-11-25 | 2021-03-12 | 北京立康生命科技有限公司 | 一种非依赖帽结构下进行细胞外转录的mRNA、呈递细胞及应用 |
| EP4063505A1 (en) | 2021-03-22 | 2022-09-28 | Fondazione Istituto Italiano di Tecnologia | Functional nucleic acid molecules directed to targets for nervous system disorders |
| GB202205423D0 (en) | 2022-04-12 | 2022-05-25 | Transine Therapeutics Ltd | Functional nucleic acid molecule |
| GB202207796D0 (en) | 2022-05-26 | 2022-07-13 | Fondazione St Italiano Tecnologia | Functional nucleic acid molecule |
| GB202207795D0 (en) | 2022-05-26 | 2022-07-13 | Fondazione St Italiano Tecnologia | Functional nucleic acid molecule |
| CN116004696B (zh) * | 2023-02-01 | 2024-03-29 | 郑州贝贝生物科技有限公司 | 可与IRES组合的3ˊUTR加茎环结构基因及其应用、mRNA表达系统 |
| WO2024177429A1 (ko) * | 2023-02-24 | 2024-08-29 | 서울시립대학교 산학협력단 | Dna 템플릿 및 이를 이용하여 제조된 mrna 백신 |
| CN117947028A (zh) * | 2023-12-29 | 2024-04-30 | 北京衡昱生物科技有限公司 | Ires-w3-31序列及其应用 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2006506984A (ja) | 2002-10-23 | 2006-03-02 | イントロン，インコーポレーテッド | 効率的なプレ−トランス−スプライシング分子の同定のためのスクリーニング方法 |
| US20070136890A1 (en) | 2003-07-03 | 2007-06-14 | Board Of Trustees Operating Michigan State University | Expression of a recombinant transgene |
| WO2012133947A1 (en) | 2011-03-30 | 2012-10-04 | Riken | Functional nucleic acid molecule and use thereof |
| JP2015535430A (ja) | 2012-11-26 | 2015-12-14 | モデルナ セラピューティクス インコーポレイテッドＭｏ | 末端修飾ｒｎａ |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6869779B1 (en) * | 1998-08-27 | 2005-03-22 | Fuso Pharmaceutical Industries, Ltd. | Nucleic acid sequence for potentiating the expression of useful gene and method therefor |
| US7468275B2 (en) * | 2000-01-28 | 2008-12-23 | The Scripps Research Institute | Synthetic internal ribosome entry sites and methods of identifying same |
| TWI300441B (en) * | 2005-09-21 | 2008-09-01 | Univ Chung Yuan Christian | A polynucleotide with ires activity |
| KR101161622B1 (ko) * | 2009-08-31 | 2012-07-04 | 헬릭스 주식회사 | 번역 효율 증진용 ｄｎａ 단편 및 이를 포함하는 재조합 벡터 |
| FI3597749T3 (fi) * | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| ES2576126T3 (es) * | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias |
| CN105624156B (zh) * | 2014-11-04 | 2021-07-16 | 清华大学 | 含有反向sineb2重复序列的人工非编码rna及其在增强靶蛋白翻译中的用途 |
| EP3350327B1 (en) * | 2015-10-23 | 2018-09-26 | Caribou Biosciences, Inc. | Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids |
-
2017
- 2017-09-20 IT IT102017000105372A patent/IT201700105372A1/it unknown
-
2018
- 2018-09-20 US US16/647,721 patent/US11649456B2/en active Active
- 2018-09-20 EP EP18782521.1A patent/EP3684928A1/en active Pending
- 2018-09-20 WO PCT/IB2018/057262 patent/WO2019058304A1/en not_active Ceased
- 2018-09-20 KR KR1020207011322A patent/KR102677300B1/ko active Active
- 2018-09-20 JP JP2020537887A patent/JP7258031B2/ja active Active
- 2018-09-20 CA CA3076163A patent/CA3076163A1/en active Pending
- 2018-09-20 CN CN201880074261.4A patent/CN111386344B/zh active Active
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2006506984A (ja) | 2002-10-23 | 2006-03-02 | イントロン，インコーポレーテッド | 効率的なプレ−トランス−スプライシング分子の同定のためのスクリーニング方法 |
| US20070136890A1 (en) | 2003-07-03 | 2007-06-14 | Board Of Trustees Operating Michigan State University | Expression of a recombinant transgene |
| WO2012133947A1 (en) | 2011-03-30 | 2012-10-04 | Riken | Functional nucleic acid molecule and use thereof |
| JP2015535430A (ja) | 2012-11-26 | 2015-12-14 | モデルナ セラピューティクス インコーポレイテッドＭｏ | 末端修飾ｒｎａ |
Non-Patent Citations (3)
| Title |
|---|
| Biochem. Soc. Trans., 2010年，Vol.38，p.1581-1586，doi:10.1042/BST0381581 |
| RNA Biology, 2015年，Vol.12, No.8，p.771-779，http://dx.doi.org/10.1080/15476286.2015.1060395 |
| トランスサインテクノロジーズ株式会社製品のSINEUPの販売を開始しました, 2013年，ニュース・イベント，（株）ダナフォーム，p.1-3，https://www.dnaform.jp/ja/information/20130320_1/, 検索日：2022年6月14日 |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3684928A1 (en) | 2020-07-29 |
| WO2019058304A1 (en) | 2019-03-28 |
| CN111386344B (zh) | 2024-02-02 |
| CA3076163A1 (en) | 2019-03-28 |
| IT201700105372A1 (it) | 2019-03-20 |
| JP2020535845A (ja) | 2020-12-10 |
| KR20200117975A (ko) | 2020-10-14 |
| US20200224197A1 (en) | 2020-07-16 |
| US11649456B2 (en) | 2023-05-16 |
| CN111386344A (zh) | 2020-07-07 |
| KR102677300B1 (ko) | 2024-06-24 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7258031B2 (ja) | 機能性核酸分子及びその使用 | |
| Buratti et al. | RNA folding affects the recruitment of SR proteins by mouse and human polypurinic enhancer elements in the fibronectin EDA exon | |
| Huang et al. | CPEB3 and CPEB4 in neurons: analysis of RNA‐binding specificity and translational control of AMPA receptor GluR2 mRNA | |
| Dumbovic et al. | Emerging roles of macrosatellite repeats in genome organization and disease development | |
| Ponicsan et al. | Genomic gems: SINE RNAs regulate mRNA production | |
| Chatterjee et al. | Role of 5′‐and 3′‐untranslated regions of mRNAs in human diseases | |
| Van Eden et al. | Demonstrating internal ribosome entry sites in eukaryotic mRNAs using stringent RNA test procedures | |
| Paek et al. | RNA-binding protein hnRNP D modulates internal ribosome entry site-dependent translation of hepatitis C virus RNA | |
| Baird et al. | A search for structurally similar cellular internal ribosome entry sites | |
| Vivinus et al. | An element within the 5′ untranslated region of human Hsp70 mRNA which acts as a general enhancer of mRNA translation | |
| Merrill et al. | Cell-type-specific repression of internal ribosome entry site activity by double-stranded RNA-binding protein 76 | |
| Terenin et al. | A cross-kingdom internal ribosome entry site reveals a simplified mode of internal ribosome entry | |
| Spriggs et al. | Canonical initiation factor requirements of the Myc family of internal ribosome entry segments | |
| Toribio et al. | An RNA trapping mechanism in Alphavirus mRNA promotes ribosome stalling and translation initiation | |
| Abaeva et al. | Attachment of ribosomal complexes and retrograde scanning during initiation on the Halastavi arva virus IRES | |
| Matsuda et al. | Determinants of initiation codon selection during translation in mammalian cells | |
| May et al. | A sequence-independent, unstructured internal ribosome entry site is responsible for internal expression of the coat protein of turnip crinkle virus | |
| Willcocks et al. | Distinct roles for the IIId2 sub-domain in pestivirus and picornavirus internal ribosome entry sites | |
| Cheng et al. | Aberrant splicing events caused by insertion of genes of interest into expression vectors | |
| Clark et al. | Conserved nucleotides within the J domain of the encephalomyocarditis virus internal ribosome entry site are required for activity and for interaction with eIF4G | |
| Pedersen et al. | Human insulin-like growth factor II leader 2 mediates internal initiation of translation | |
| EP1002082B1 (en) | Transcriptional silencer protein nrf | |
| Okada et al. | Dissection of the beta-globin replication-initiation region reveals specific requirements for replicator elements during gene amplification | |
| Nair et al. | Sizing, stabilising, and cloning repeat-expansions for gene targeting constructs | |
| Krivtsova et al. | Aberrant expression of alternative isoforms of transcription factors in hepatocellular carcinoma |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200630 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210727 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20210727 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20220712 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20221011 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20221227 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20230307 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20230404 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 7258031 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |