GB2590880A - RNA and DNA base editing via engineered ADAR recruitment - Google Patents
RNA and DNA base editing via engineered ADAR recruitment Download PDFInfo
- Publication number
- GB2590880A GB2590880A GB2104889.7A GB202104889A GB2590880A GB 2590880 A GB2590880 A GB 2590880A GB 202104889 A GB202104889 A GB 202104889A GB 2590880 A GB2590880 A GB 2590880A
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- GB
- United Kingdom
- Prior art keywords
- nucleic acid
- vector
- domain
- rna
- sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 230000007115 recruitment Effects 0.000 title 1
- 101000865408 Homo sapiens Double-stranded RNA-specific adenosine deaminase Proteins 0.000 claims abstract 34
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims abstract 32
- 102100029791 Double-stranded RNA-specific adenosine deaminase Human genes 0.000 claims abstract 30
- 102100038191 Double-stranded RNA-specific editase 1 Human genes 0.000 claims abstract 4
- 101000742223 Homo sapiens Double-stranded RNA-specific editase 1 Proteins 0.000 claims abstract 4
- 230000035772 mutation Effects 0.000 claims abstract 4
- 108020004999 messenger RNA Proteins 0.000 claims abstract 3
- 102000039446 nucleic acids Human genes 0.000 claims 77
- 108020004707 nucleic acids Proteins 0.000 claims 77
- 150000007523 nucleic acids Chemical class 0.000 claims 77
- 239000013598 vector Substances 0.000 claims 73
- 238000010357 RNA editing Methods 0.000 claims 52
- 230000026279 RNA modification Effects 0.000 claims 52
- 238000000034 method Methods 0.000 claims 32
- 108091033319 polynucleotide Proteins 0.000 claims 21
- 102000040430 polynucleotide Human genes 0.000 claims 21
- 239000002157 polynucleotide Substances 0.000 claims 21
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 14
- 201000010099 disease Diseases 0.000 claims 13
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 102000004169 proteins and genes Human genes 0.000 claims 12
- 108091023043 Alu Element Proteins 0.000 claims 11
- 230000000295 complement effect Effects 0.000 claims 11
- 230000000692 anti-sense effect Effects 0.000 claims 8
- 230000027455 binding Effects 0.000 claims 8
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 102000043770 human ADAR Human genes 0.000 claims 4
- 238000002347 injection Methods 0.000 claims 4
- 239000007924 injection Substances 0.000 claims 4
- 239000013603 viral vector Substances 0.000 claims 4
- 108010079649 APOBEC-1 Deaminase Proteins 0.000 claims 3
- 108010004483 APOBEC-3G Deaminase Proteins 0.000 claims 3
- 101710095342 Apolipoprotein B Proteins 0.000 claims 3
- 102100040202 Apolipoprotein B-100 Human genes 0.000 claims 3
- 102100040399 C->U-editing enzyme APOBEC-2 Human genes 0.000 claims 3
- 101710132601 Capsid protein Proteins 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 3
- 102100040263 DNA dC->dU-editing enzyme APOBEC-3A Human genes 0.000 claims 3
- 102100040262 DNA dC->dU-editing enzyme APOBEC-3B Human genes 0.000 claims 3
- 102100040261 DNA dC->dU-editing enzyme APOBEC-3C Human genes 0.000 claims 3
- 102100040264 DNA dC->dU-editing enzyme APOBEC-3D Human genes 0.000 claims 3
- 102100040266 DNA dC->dU-editing enzyme APOBEC-3F Human genes 0.000 claims 3
- 102100038076 DNA dC->dU-editing enzyme APOBEC-3G Human genes 0.000 claims 3
- 102100038050 DNA dC->dU-editing enzyme APOBEC-3H Human genes 0.000 claims 3
- 101710082737 DNA dC->dU-editing enzyme APOBEC-3H Proteins 0.000 claims 3
- 101000964322 Homo sapiens C->U-editing enzyme APOBEC-2 Proteins 0.000 claims 3
- 101000964378 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3A Proteins 0.000 claims 3
- 101000964385 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3B Proteins 0.000 claims 3
- 101000964383 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3C Proteins 0.000 claims 3
- 101000964382 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3D Proteins 0.000 claims 3
- 101000964377 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3F Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 230000003197 catalytic effect Effects 0.000 claims 3
- 230000017156 mRNA modification Effects 0.000 claims 3
- 201000006938 muscular dystrophy Diseases 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 230000008685 targeting Effects 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 102100040397 C->U-editing enzyme APOBEC-1 Human genes 0.000 claims 2
- 101710197658 Capsid protein VP1 Proteins 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 102100024692 Double-stranded RNA-specific editase B2 Human genes 0.000 claims 2
- 101000686486 Homo sapiens Double-stranded RNA-specific editase B2 Proteins 0.000 claims 2
- 208000021642 Muscular disease Diseases 0.000 claims 2
- 208000022873 Ocular disease Diseases 0.000 claims 2
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 101710108545 Viral protein 1 Proteins 0.000 claims 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 238000007385 chemical modification Methods 0.000 claims 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 239000002502 liposome Substances 0.000 claims 2
- 208000030159 metabolic disease Diseases 0.000 claims 2
- 239000002105 nanoparticle Substances 0.000 claims 2
- 208000015122 neurodegenerative disease Diseases 0.000 claims 2
- 102000044158 nucleic acid binding protein Human genes 0.000 claims 2
- 108700020942 nucleic acid binding protein Proteins 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 238000012163 sequencing technique Methods 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 238000002560 therapeutic procedure Methods 0.000 claims 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 102000012758 APOBEC-1 Deaminase Human genes 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 1
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 claims 1
- 101710094648 Coat protein Proteins 0.000 claims 1
- 230000004568 DNA-binding Effects 0.000 claims 1
- 108090000079 Glucocorticoid Receptors Proteins 0.000 claims 1
- 102100033417 Glucocorticoid receptor Human genes 0.000 claims 1
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 claims 1
- 101000825762 Homo sapiens Histone RNA hairpin-binding protein Proteins 0.000 claims 1
- 101000800426 Homo sapiens Putative C->U-editing enzyme APOBEC-4 Proteins 0.000 claims 1
- 229930010555 Inosine Natural products 0.000 claims 1
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 1
- 208000035719 Maculopathy Diseases 0.000 claims 1
- 101710125418 Major capsid protein Proteins 0.000 claims 1
- 101710141454 Nucleoprotein Proteins 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 101710083689 Probable capsid protein Proteins 0.000 claims 1
- 102100033091 Putative C->U-editing enzyme APOBEC-4 Human genes 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 208000006289 Rett Syndrome Diseases 0.000 claims 1
- 102400000700 Tumor necrosis factor, membrane form Human genes 0.000 claims 1
- 101800000716 Tumor necrosis factor, membrane form Proteins 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 229960005305 adenosine Drugs 0.000 claims 1
- 210000004900 c-terminal fragment Anatomy 0.000 claims 1
- 238000010367 cloning Methods 0.000 claims 1
- 229940104302 cytosine Drugs 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000010255 intramuscular injection Methods 0.000 claims 1
- 239000007927 intramuscular injection Substances 0.000 claims 1
- 239000007928 intraperitoneal injection Substances 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 210000004898 n-terminal fragment Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000002062 proliferating effect Effects 0.000 claims 1
- 230000002685 pulmonary effect Effects 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 238000010254 subcutaneous injection Methods 0.000 claims 1
- 239000007929 subcutaneous injection Substances 0.000 claims 1
- 229940113082 thymine Drugs 0.000 claims 1
- 230000002463 transducing effect Effects 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
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- 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
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- A—HUMAN NECESSITIES
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
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- C12N2320/31—Combination therapy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/33—Alteration of splicing
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14341—Use of virus, viral particle or viral elements as a vector
- C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862728007P | 2018-09-06 | 2018-09-06 | |
| US201862766433P | 2018-10-17 | 2018-10-17 | |
| US201862773150P | 2018-11-29 | 2018-11-29 | |
| US201862773146P | 2018-11-29 | 2018-11-29 | |
| US201862780241P | 2018-12-15 | 2018-12-15 | |
| PCT/US2019/050095 WO2020051555A1 (en) | 2018-09-06 | 2019-09-06 | Rna and dna base editing via engneered adar recruitment |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| GB202104889D0 GB202104889D0 (en) | 2021-05-19 |
| GB2590880A true GB2590880A (en) | 2021-07-07 |
Family
ID=69723301
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| GB2104889.7A Withdrawn GB2590880A (en) | 2018-09-06 | 2019-09-06 | RNA and DNA base editing via engineered ADAR recruitment |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20220010333A1 (OSRAM) |
| EP (2) | EP3847255A4 (OSRAM) |
| JP (1) | JP7720623B6 (OSRAM) |
| CN (2) | CN119193702A (OSRAM) |
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| CN118416088A (zh) * | 2017-03-03 | 2024-08-02 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| WO2019104094A2 (en) | 2017-11-21 | 2019-05-31 | The Regents Of The University Of California | Fusion proteins and methods for site-directed genome editing |
| PH12021552613A1 (en) | 2019-04-15 | 2022-06-27 | Edigene Therapeutics Beijing Inc | METHODS AND COMPOSITIONS FOR EDITING RNAs |
| CN114269919B (zh) * | 2019-07-12 | 2023-08-18 | 北京大学 | 采用工程化rna利用内源adar进行靶向的rna编辑 |
| US20230036370A1 (en) * | 2019-12-02 | 2023-02-02 | The Regents Of The University Of California | Engineering circular guide rnas |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| JP7754721B2 (ja) * | 2019-12-09 | 2025-10-15 | アステラス製薬株式会社 | 標的ｒｎａを編集するための機能性領域を付加したアンチセンス型ガイドｒｎａ |
| WO2021216853A1 (en) * | 2020-04-22 | 2021-10-28 | Shape Therapeutics Inc. | Compositions and methods using snrna components |
| WO2021242870A1 (en) * | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | Compositions and methods for genome editing |
| JP2023528039A (ja) * | 2020-05-26 | 2023-07-03 | シェイプ セラピューティクス インコーポレイテッド | 改変環状ポリヌクレオチド |
| EP4158019A1 (en) * | 2020-05-26 | 2023-04-05 | Shape Therapeutics Inc. | Methods and compositions relating to engineered guide systems for adenosine deaminase acting on rna editing |
| JP2023532375A (ja) * | 2020-07-06 | 2023-07-27 | 北京▲輯▼因医▲療▼科技有限公司 | 改良されたｒｎａ編集方法 |
| CA3190477A1 (en) * | 2020-07-30 | 2022-02-03 | Adarx Pharmaceuticals, Inc. | Adar dependent editing compositions and methods of use thereof |
| CN115989041A (zh) * | 2020-08-24 | 2023-04-18 | 波涛生命科学有限公司 | 经工程化为表达adar1的细胞和非人动物及其用途 |
| WO2022078569A1 (en) * | 2020-10-12 | 2022-04-21 | Eberhard Karls Universität Tübingen | Artificial nucleic acids for rna editing |
| WO2022103852A1 (en) * | 2020-11-11 | 2022-05-19 | Shape Therapeutics Inc. | Rna-editing compositions and methods of use |
| WO2022119975A2 (en) * | 2020-12-01 | 2022-06-09 | Shape Therapeutics Inc. | High throughput rna-editing screening methods |
| US20240150754A1 (en) * | 2020-12-25 | 2024-05-09 | Astellas Pharma Inc. | Guide rna for editing polyadenylation signal sequence of target rna |
| WO2022251097A1 (en) * | 2021-05-25 | 2022-12-01 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| US20240279656A1 (en) * | 2021-06-29 | 2024-08-22 | Shape Therapeutics Inc. | Engineered Guide RNAs and Polynucleotides |
| US20250001011A1 (en) * | 2021-08-18 | 2025-01-02 | Peking University | Engineered adar-recruiting rnas and methods of use thereof |
| IL311101A (en) | 2021-09-03 | 2024-04-01 | Tacit Therapeutics Inc | RNA editing through recruitment of SPLICEOSOME components |
| JP2023040852A (ja) * | 2021-09-10 | 2023-03-23 | 隆光 矢野 | －１フレームシフトを誘導するための１本鎖核酸分子及び組成物 |
| US20230167441A1 (en) * | 2021-11-30 | 2023-06-01 | Minghong Zhong | Segmented Nucleic Acids |
| US20250115908A1 (en) * | 2022-02-07 | 2025-04-10 | The Regents Of The University Of California | Method of interfering with repetitive rna |
| US20250215426A1 (en) * | 2022-04-05 | 2025-07-03 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of muscular dystrophies |
| CN116732039B (zh) * | 2023-08-03 | 2023-11-14 | 呈诺再生医学科技(北京)有限公司 | 一种促进rna序列在细胞内直接环化翻译的序列组合及其应用 |
| CN117210435A (zh) * | 2023-08-29 | 2023-12-12 | 苏州湃芮生物科技有限公司 | 一种用于调控rna甲基化修饰的编辑系统及其应用 |
| CN121059628A (zh) * | 2024-06-03 | 2025-12-05 | 时夕(广州)生物科技有限公司 | 调控RNA剪接的向导agRNA |
| WO2026080897A1 (en) * | 2024-10-11 | 2026-04-16 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of chronic pain |
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| KR102057540B1 (ko) * | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| ES2810375T3 (es) * | 2014-12-17 | 2021-03-08 | Proqr Therapeutics Ii Bv | Edición direccionada de ARN |
| JP2018538288A (ja) * | 2015-12-14 | 2018-12-27 | コールド スプリング ハーバー ラボラトリー | アラジール症候群の処置のためのアンチセンスオリゴマー |
| CA3062595A1 (en) * | 2017-05-10 | 2018-11-15 | The Regents Of The University Of California | Directed editing of cellular rna via nuclear delivery of crispr/cas9 |
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- 2019-09-06 EP EP19857915.3A patent/EP3847255A4/en not_active Withdrawn
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- 2019-09-06 CN CN202410647165.1A patent/CN119193702A/zh active Pending
- 2019-09-06 JP JP2021511542A patent/JP7720623B6/ja active Active
- 2019-09-06 WO PCT/US2019/050095 patent/WO2020051555A1/en not_active Ceased
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2025
- 2025-07-07 US US19/261,672 patent/US20260092292A1/en active Pending
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| WO2017219027A1 (en) * | 2016-06-17 | 2017-12-21 | The Broad Institute Inc. | Type vi crispr orthologs and systems |
| WO2017220751A1 (en) * | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
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| EP4332224A2 (en) | 2024-03-06 |
| AU2019336793B2 (en) | 2024-11-21 |
| CA3110998A1 (en) | 2020-03-12 |
| US20260092292A1 (en) | 2026-04-02 |
| CN119193702A (zh) | 2024-12-27 |
| GB202104889D0 (en) | 2021-05-19 |
| JP7720623B6 (ja) | 2025-09-18 |
| EP3847255A4 (en) | 2024-01-10 |
| CN112996912A (zh) | 2021-06-18 |
| WO2020051555A1 (en) | 2020-03-12 |
| EP3847255A1 (en) | 2021-07-14 |
| AU2019336793A1 (en) | 2021-04-08 |
| US20220010333A1 (en) | 2022-01-13 |
| JP2021536244A (ja) | 2021-12-27 |
| JP7720623B2 (ja) | 2025-08-08 |
| EP4332224A3 (en) | 2024-06-05 |
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