JP2020535803A5 - - Google Patents

Info

Publication number

JP2020535803A5

JP2020535803A5 JP2020516666A JP2020516666A JP2020535803A5 JP 2020535803 A5 JP2020535803 A5 JP 2020535803A5 JP 2020516666 A JP2020516666 A JP 2020516666A JP 2020516666 A JP2020516666 A JP 2020516666A JP 2020535803 A5 JP2020535803 A5 JP 2020535803A5

Authority

JP

Japan

Prior art keywords

virus

vector

nucleic acid

promoter

rnai

Prior art date

2018-09-21

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.)

Granted

Links

• Espacenet
• Global Dossier
• Discuss

• 239000002245 particle Substances 0.000 claims 29
• 150000007523 nucleic acids Chemical class 0.000 claims 25
• 239000013598 vector Substances 0.000 claims 21
• 241000700605 Viruses Species 0.000 claims 20
• 108091030071 RNAI Proteins 0.000 claims 19
• 230000009368 gene silencing by RNA Effects 0.000 claims 19
• 241000702421 Dependoparvovirus Species 0.000 claims 17
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 16
• 241000701161 unidentified adenovirus Species 0.000 claims 10
• 108020004707 nucleic acids Proteins 0.000 claims 9
• 102000039446 nucleic acids Human genes 0.000 claims 9
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims 8
• 210000000234 capsid Anatomy 0.000 claims 8
• 239000013608 rAAV vector Substances 0.000 claims 7
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 6
• 241000713666 Lentivirus Species 0.000 claims 6
• 210000002966 serum Anatomy 0.000 claims 5
• 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
• 241000283690 Bos taurus Species 0.000 claims 4
• 208000023105 Huntington disease Diseases 0.000 claims 4
• 239000002773 nucleotide Substances 0.000 claims 4
• 125000003729 nucleotide group Chemical group 0.000 claims 4
• 230000008488 polyadenylation Effects 0.000 claims 4
• 239000004055 small Interfering RNA Substances 0.000 claims 4
• 241000282465 Canis Species 0.000 claims 3
• 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 3
• 108700011259 MicroRNAs Proteins 0.000 claims 3
• 239000002679 microRNA Substances 0.000 claims 3
• 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 claims 2
• 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
• 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
• 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
• 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
• 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
• 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
• 241000701022 Cytomegalovirus Species 0.000 claims 2
• 241001115402 Ebolavirus Species 0.000 claims 2
• 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
• 241000124008 Mammalia Species 0.000 claims 2
• 241001115401 Marburgvirus Species 0.000 claims 2
• 241001494479 Pecora Species 0.000 claims 2
• 108091027967 Small hairpin RNA Proteins 0.000 claims 2
• 108020004459 Small interfering RNA Proteins 0.000 claims 2
• 230000000527 lymphocytic effect Effects 0.000 claims 2
• 229920001184 polypeptide Polymers 0.000 claims 2
• 102000004196 processed proteins & peptides Human genes 0.000 claims 2
• 108090000765 processed proteins & peptides Proteins 0.000 claims 2
• 208000003265 stomatitis Diseases 0.000 claims 2
• 241000283707 Capra Species 0.000 claims 1
• 208000010772 Dog disease Diseases 0.000 claims 1
• 206010066919 Epidemic polyarthritis Diseases 0.000 claims 1
• 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
• 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
• 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims 1
• 241000713869 Moloney murine leukemia virus Species 0.000 claims 1
• 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
• 241000710942 Ross River virus Species 0.000 claims 1
• 206010039491 Sarcoma Diseases 0.000 claims 1
• 108010017842 Telomerase Proteins 0.000 claims 1
• 239000004098 Tetracycline Substances 0.000 claims 1
• 108010022394 Threonine synthase Proteins 0.000 claims 1
• 108010006025 bovine growth hormone Proteins 0.000 claims 1
• 230000000295 complement effect Effects 0.000 claims 1
• 102000004419 dihydrofolate reductase Human genes 0.000 claims 1
• 239000003937 drug carrier Substances 0.000 claims 1
• 239000003623 enhancer Substances 0.000 claims 1
• 239000013604 expression vector Substances 0.000 claims 1
• 230000001697 htt accumulation Effects 0.000 claims 1
• 230000002401 inhibitory effect Effects 0.000 claims 1
• 210000004185 liver Anatomy 0.000 claims 1
• 230000035772 mutation Effects 0.000 claims 1
• 239000013646 rAAV2 vector Substances 0.000 claims 1
• 230000001177 retroviral effect Effects 0.000 claims 1
• 229960002180 tetracycline Drugs 0.000 claims 1
• 229930101283 tetracycline Natural products 0.000 claims 1
• 235000019364 tetracycline Nutrition 0.000 claims 1
• 150000003522 tetracyclines Chemical class 0.000 claims 1
• 241001529453 unidentified herpesvirus Species 0.000 claims 1
• 238000011144 upstream manufacturing Methods 0.000 claims 1
• 230000003612 virological effect Effects 0.000 claims 1