JPWO2020132115A5 - - Google Patents

Info

Publication number

JPWO2020132115A5

JPWO2020132115A5 JP2021534702A JP2021534702A JPWO2020132115A5 JP WO2020132115 A5 JPWO2020132115 A5 JP WO2020132115A5 JP 2021534702 A JP2021534702 A JP 2021534702A JP 2021534702 A JP2021534702 A JP 2021534702A JP WO2020132115 A5 JPWO2020132115 A5 JP WO2020132115A5

Authority

JP

Japan

Prior art keywords

sequence

raav

nucleic acid

composition

acid molecule

Prior art date

2022-05-20

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 44
• 108020004707 nucleic acids Proteins 0.000 claims description 38
• 102000039446 nucleic acids Human genes 0.000 claims description 38
• 101000930910 Homo sapiens Glucose-6-phosphatase catalytic subunit 1 Proteins 0.000 claims description 34
• 102100036264 Glucose-6-phosphatase catalytic subunit 1 Human genes 0.000 claims description 32
• 239000013598 vector Substances 0.000 claims description 29
• 238000000034 method Methods 0.000 claims description 24
• 108091023043 Alu Element Proteins 0.000 claims description 21
• 239000002773 nucleotide Substances 0.000 claims description 17
• 125000003729 nucleotide group Chemical group 0.000 claims description 17
• 230000014509 gene expression Effects 0.000 claims description 13
• 108010076504 Protein Sorting Signals Proteins 0.000 claims description 12
• 108091026890 Coding region Proteins 0.000 claims description 10
• 206010018464 Glycogen storage disease type I Diseases 0.000 claims description 10
• 241000282414 Homo sapiens Species 0.000 claims description 10
• 208000011460 glycogen storage disease due to glucose-6-phosphatase deficiency type IA Diseases 0.000 claims description 10
• 239000003623 enhancer Substances 0.000 claims description 9
• 239000013607 AAV vector Substances 0.000 claims description 8
• 210000000234 capsid Anatomy 0.000 claims description 8
• 230000008488 polyadenylation Effects 0.000 claims description 8
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 7
• 210000004027 cell Anatomy 0.000 claims description 7
• 241000702421 Dependoparvovirus Species 0.000 claims description 5
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
• 239000003937 drug carrier Substances 0.000 claims description 4
• 108091028043 Nucleic acid sequence Proteins 0.000 claims description 3
• 238000004113 cell culture Methods 0.000 claims description 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 description 2
• 210000003527 eukaryotic cell Anatomy 0.000 claims description 2
• 238000003306 harvesting Methods 0.000 claims description 2
• 239000013647 rAAV8 vector Substances 0.000 claims description 2
• 238000001990 intravenous administration Methods 0.000 claims 2
• 238000004519 manufacturing process Methods 0.000 claims 1
• 241000700605 Viruses Species 0.000 description 4
• 239000013603 viral vector Substances 0.000 description 4
• 238000012217 deletion Methods 0.000 description 3
• 230000037430 deletion Effects 0.000 description 3
• 230000000694 effects Effects 0.000 description 3
• 101100335761 Homo sapiens G6PC1 gene Proteins 0.000 description 2
• 210000004185 liver Anatomy 0.000 description 2
• 108700028146 Genetic Enhancer Elements Proteins 0.000 description 1
• 241000124008 Mammalia Species 0.000 description 1
• 241001494479 Pecora Species 0.000 description 1
• 241000283984 Rodentia Species 0.000 description 1
• 238000001415 gene therapy Methods 0.000 description 1
• 210000003734 kidney Anatomy 0.000 description 1
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• 239000002245 particle Substances 0.000 description 1
• 230000001177 retroviral effect Effects 0.000 description 1