JPWO2020041498A5 - - Google Patents

Info

Publication number

JPWO2020041498A5

JPWO2020041498A5 JP2021509223A JP2021509223A JPWO2020041498A5 JP WO2020041498 A5 JPWO2020041498 A5 JP WO2020041498A5 JP 2021509223 A JP2021509223 A JP 2021509223A JP 2021509223 A JP2021509223 A JP 2021509223A JP WO2020041498 A5 JPWO2020041498 A5 JP WO2020041498A5

Authority

JP

Japan

Prior art keywords

sequence

aav

gpr108

seq

polypeptide

Prior art date

2022-07-25

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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• 238000000034 method Methods 0.000 claims description 54
• 101001070470 Homo sapiens Protein GPR108 Proteins 0.000 claims description 33
• 102100034142 Protein GPR108 Human genes 0.000 claims description 33
• 229920001184 polypeptide Polymers 0.000 claims description 33
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 33
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 33
• 239000000203 mixture Substances 0.000 claims description 14
• 230000026683 transduction Effects 0.000 claims description 14
• 238000010361 transduction Methods 0.000 claims description 14
• 239000003814 drug Substances 0.000 claims description 12
• 229940124597 therapeutic agent Drugs 0.000 claims description 12
• 210000000234 capsid Anatomy 0.000 claims description 10
• 241000702421 Dependoparvovirus Species 0.000 claims description 8
• 230000001419 dependent effect Effects 0.000 claims description 8
• 108090000623 proteins and genes Proteins 0.000 claims description 8
• 102000004169 proteins and genes Human genes 0.000 claims description 8
• 150000001413 amino acids Chemical class 0.000 claims description 6
• 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 5
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
• 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 5
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 5
• 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 5
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 5
• 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 4
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 4
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
• 239000011230 binding agent Substances 0.000 claims description 4
• 108091023037 Aptamer Proteins 0.000 claims description 2
• 210000004027 cell Anatomy 0.000 description 18
• 150000001875 compounds Chemical class 0.000 description 4
• 108091032973 (ribonucleotides)n+m Proteins 0.000 description 2
• 230000000694 effects Effects 0.000 description 2
• 230000002452 interceptive effect Effects 0.000 description 2
• 108091030071 RNAI Proteins 0.000 description 1
• 108020004459 Small interfering RNA Proteins 0.000 description 1
• 108700019146 Transgenes Proteins 0.000 description 1
• 210000002798 bone marrow cell Anatomy 0.000 description 1
• 210000002889 endothelial cell Anatomy 0.000 description 1
• 210000002919 epithelial cell Anatomy 0.000 description 1
• 230000009368 gene silencing by RNA Effects 0.000 description 1
• 210000002064 heart cell Anatomy 0.000 description 1
• 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
• 210000003494 hepatocyte Anatomy 0.000 description 1
• 238000001727 in vivo Methods 0.000 description 1
• 230000003993 interaction Effects 0.000 description 1
• 210000003292 kidney cell Anatomy 0.000 description 1
• 210000005265 lung cell Anatomy 0.000 description 1
• 239000000463 material Substances 0.000 description 1