JPWO2019153009A5 - - Google Patents

Info

Publication number

JPWO2019153009A5

JPWO2019153009A5 JP2020564045A JP2020564045A JPWO2019153009A5 JP WO2019153009 A5 JPWO2019153009 A5 JP WO2019153009A5 JP 2020564045 A JP2020564045 A JP 2020564045A JP 2020564045 A JP2020564045 A JP 2020564045A JP WO2019153009 A5 JPWO2019153009 A5 JP WO2019153009A5

Authority

JP

Japan

Prior art keywords

nucleic acid

acid sequence

regulatory element

segment

seq

Prior art date

2022-02-09

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
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• 150000007523 nucleic acids Chemical class 0.000 claims description 310
• 108091028043 Nucleic acid sequence Proteins 0.000 claims description 173
• 108020004707 nucleic acids Proteins 0.000 claims description 137
• 102000039446 nucleic acids Human genes 0.000 claims description 137
• 230000001105 regulatory effect Effects 0.000 claims description 122
• 239000013598 vector Substances 0.000 claims description 36
• 108700019146 Transgenes Proteins 0.000 claims description 18
• 239000003623 enhancer Substances 0.000 claims description 15
• 239000000203 mixture Substances 0.000 claims description 15
• 210000004027 cell Anatomy 0.000 claims description 14
• 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 description 10
• 238000000034 method Methods 0.000 claims description 8
• 102100036912 Desmin Human genes 0.000 claims description 4
• 108010044052 Desmin Proteins 0.000 claims description 4
• 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims description 4
• 102000001435 Synapsin Human genes 0.000 claims description 4
• 108050009621 Synapsin Proteins 0.000 claims description 4
• 241000700605 Viruses Species 0.000 claims description 4
• 210000005045 desmin Anatomy 0.000 claims description 4
• 210000001808 exosome Anatomy 0.000 claims description 4
• 239000013646 rAAV2 vector Substances 0.000 claims description 4
• 239000013603 viral vector Substances 0.000 claims description 4
• 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
• 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
• 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
• 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
• 102100029470 Apolipoprotein E Human genes 0.000 claims description 2
• 101710095339 Apolipoprotein E Proteins 0.000 claims description 2
• 241000702421 Dependoparvovirus Species 0.000 claims description 2
• 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims description 2
• 206010053185 Glycogen storage disease type II Diseases 0.000 claims description 2
• 241000713666 Lentivirus Species 0.000 claims description 2
• 241000700618 Vaccinia virus Species 0.000 claims description 2
• 230000002378 acidificating effect Effects 0.000 claims description 2
• 108010028144 alpha-Glucosidases Proteins 0.000 claims description 2
• 239000000412 dendrimer Substances 0.000 claims description 2
• 229920000736 dendritic polymer Polymers 0.000 claims description 2
• 201000004502 glycogen storage disease II Diseases 0.000 claims description 2
• 210000003494 hepatocyte Anatomy 0.000 claims description 2
• 239000002502 liposome Substances 0.000 claims description 2
• 210000004185 liver Anatomy 0.000 claims description 2
• 210000000663 muscle cell Anatomy 0.000 claims description 2
• 239000002105 nanoparticle Substances 0.000 claims description 2
• 210000002569 neuron Anatomy 0.000 claims description 2
• 238000013518 transcription Methods 0.000 claims description 2
• 230000035897 transcription Effects 0.000 claims description 2
• 241000701161 unidentified adenovirus Species 0.000 claims description 2
• 241000701447 unidentified baculovirus Species 0.000 claims description 2
• 241001529453 unidentified herpesvirus Species 0.000 claims description 2
• 241001430294 unidentified retrovirus Species 0.000 claims description 2
• 239000008194 pharmaceutical composition Substances 0.000 claims 1