JP2020519294A5 - - Google Patents

Info

Publication number

JP2020519294A5

JP2020519294A5 JP2019562556A JP2019562556A JP2020519294A5 JP 2020519294 A5 JP2020519294 A5 JP 2020519294A5 JP 2019562556 A JP2019562556 A JP 2019562556A JP 2019562556 A JP2019562556 A JP 2019562556A JP 2020519294 A5 JP2020519294 A5 JP 2020519294A5

Authority

JP

Japan

Prior art keywords

nucleic acid

optionally

plasmid

host cell

transgene

Prior art date

2018-05-11

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

Pending

Links

• Espacenet
• Global Dossier
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• 108020004707 nucleic acids Proteins 0.000 claims 18
• 102000039446 nucleic acids Human genes 0.000 claims 18
• 150000007523 nucleic acids Chemical class 0.000 claims 18
• 210000004027 cell Anatomy 0.000 claims 16
• 238000000034 method Methods 0.000 claims 16
• 239000013612 plasmid Substances 0.000 claims 11
• 108091070501 miRNA Proteins 0.000 claims 9
• 239000002679 microRNA Substances 0.000 claims 9
• 108700019146 Transgenes Proteins 0.000 claims 8
• 239000013607 AAV vector Substances 0.000 claims 3
• 238000004806 packaging method and process Methods 0.000 claims 3
• 239000013598 vector Substances 0.000 claims 3
• 108091026821 Artificial microRNA Proteins 0.000 claims 2
• 241000238631 Hexapoda Species 0.000 claims 2
• 210000005260 human cell Anatomy 0.000 claims 2
• 230000002401 inhibitory effect Effects 0.000 claims 2
• 230000002452 interceptive effect Effects 0.000 claims 2
• 239000013603 viral vector Substances 0.000 claims 2
• 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 1
• 108020003589 5' Untranslated Regions Proteins 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
• 241001634120 Adeno-associated virus - 5 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
• 108020004705 Codon Proteins 0.000 claims 1
• 241000702421 Dependoparvovirus Species 0.000 claims 1
• 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
• 241000713666 Lentivirus Species 0.000 claims 1
• 108091027967 Small hairpin RNA Proteins 0.000 claims 1
• 241000256251 Spodoptera frugiperda Species 0.000 claims 1
• 241000700605 Viruses Species 0.000 claims 1
• 231100000433 cytotoxic Toxicity 0.000 claims 1
• 230000001472 cytotoxic effect Effects 0.000 claims 1
• 230000001627 detrimental effect Effects 0.000 claims 1
• 230000005764 inhibitory process Effects 0.000 claims 1
• 210000004962 mammalian cell Anatomy 0.000 claims 1
• 108091024102 miR856 stem-loop Proteins 0.000 claims 1
• 239000002245 particle Substances 0.000 claims 1
• 230000002085 persistent effect Effects 0.000 claims 1
• 108090000623 proteins and genes Proteins 0.000 claims 1
• 102000004169 proteins and genes Human genes 0.000 claims 1
• 230000003362 replicative effect Effects 0.000 claims 1
• 230000001177 retroviral effect Effects 0.000 claims 1
• 239000004055 small Interfering RNA Substances 0.000 claims 1
• 230000001052 transient effect Effects 0.000 claims 1
• 241000701161 unidentified adenovirus Species 0.000 claims 1
• 230000003612 virological effect Effects 0.000 claims 1