JP2021500070A5 - - Google Patents

Info

Publication number

JP2021500070A5

JP2021500070A5 JP2020542536A JP2020542536A JP2021500070A5 JP 2021500070 A5 JP2021500070 A5 JP 2021500070A5 JP 2020542536 A JP2020542536 A JP 2020542536A JP 2020542536 A JP2020542536 A JP 2020542536A JP 2021500070 A5 JP2021500070 A5 JP 2021500070A5

Authority

JP

Japan

Prior art keywords

amino acid

seq

capsid protein

protein corresponding

aav

Prior art date

2018-10-17

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
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• 150000001413 amino acids Chemical class 0.000 claims 202
• 108090000565 Capsid Proteins Proteins 0.000 claims 108
• 102100023321 Ceruloplasmin Human genes 0.000 claims 108
• 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 102
• 239000002773 nucleotide Substances 0.000 claims 43
• 125000003729 nucleotide group Chemical group 0.000 claims 43
• 238000000034 method Methods 0.000 claims 18
• 108090000623 proteins and genes Proteins 0.000 claims 17
• 108091026890 Coding region Proteins 0.000 claims 12
• 230000037430 deletion Effects 0.000 claims 11
• 238000012217 deletion Methods 0.000 claims 11
• 101150013707 HBB gene Proteins 0.000 claims 9
• 125000003275 alpha amino acid group Chemical group 0.000 claims 9
• 210000004027 cell Anatomy 0.000 claims 9
• 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 8
• 101710163270 Nuclease Proteins 0.000 claims 8
• 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 7
• 230000002950 deficient Effects 0.000 claims 7
• 230000037431 insertion Effects 0.000 claims 7
• 238000003780 insertion Methods 0.000 claims 7
• 230000010076 replication Effects 0.000 claims 7
• 241000702421 Dependoparvovirus Species 0.000 claims 6
• 210000000234 capsid Anatomy 0.000 claims 6
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
• USWINTIHFQKJTR-UHFFFAOYSA-N 3-hydroxynaphthalene-2,7-disulfonic acid Chemical compound C1=C(S(O)(=O)=O)C=C2C=C(S(O)(=O)=O)C(O)=CC2=C1 USWINTIHFQKJTR-UHFFFAOYSA-N 0.000 claims 5
• 238000010361 transduction Methods 0.000 claims 5
• 241000255925 Diptera Species 0.000 claims 4
• 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 4
• 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 4
• 210000003958 hematopoietic stem cell Anatomy 0.000 claims 4
• 230000035772 mutation Effects 0.000 claims 4
• 210000000130 stem cell Anatomy 0.000 claims 4
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
• -1 SEQ ID NO: 2 amino acid Chemical class 0.000 claims 3
• 239000002253 acid Substances 0.000 claims 3
• 201000010099 disease Diseases 0.000 claims 3
• 208000035475 disorder Diseases 0.000 claims 3
• 238000004806 packaging method and process Methods 0.000 claims 3
• 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 3
• 102000004169 proteins and genes Human genes 0.000 claims 3
• 230000026683 transduction Effects 0.000 claims 3
• 206010064571 Gene mutation Diseases 0.000 claims 2
• 108091081024 Start codon Proteins 0.000 claims 2
• 238000011260 co-administration Methods 0.000 claims 2
• 210000000805 cytoplasm Anatomy 0.000 claims 2
• 210000003743 erythrocyte Anatomy 0.000 claims 2
• 238000000338 in vitro Methods 0.000 claims 2
• 230000010354 integration Effects 0.000 claims 2
• 210000001778 pluripotent stem cell Anatomy 0.000 claims 2
• MTJGVAJYTOXFJH-UHFFFAOYSA-N 3-aminonaphthalene-1,5-disulfonic acid Chemical group C1=CC=C(S(O)(=O)=O)C2=CC(N)=CC(S(O)(=O)=O)=C21 MTJGVAJYTOXFJH-UHFFFAOYSA-N 0.000 claims 1
• HBZVNWNSRNTWPS-UHFFFAOYSA-N 6-amino-4-hydroxynaphthalene-2-sulfonic acid Chemical compound C1=C(S(O)(=O)=O)C=C(O)C2=CC(N)=CC=C21 HBZVNWNSRNTWPS-UHFFFAOYSA-N 0.000 claims 1
• 108700028369 Alleles Proteins 0.000 claims 1
• 108020004705 Codon Proteins 0.000 claims 1
• 102100031780 Endonuclease Human genes 0.000 claims 1
• 108010042407 Endonucleases Proteins 0.000 claims 1
• 108700024394 Exon Proteins 0.000 claims 1
• 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
• 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 claims 1
• 108700026244 Open Reading Frames Proteins 0.000 claims 1
• 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 claims 1
• 108020005067 RNA Splice Sites Proteins 0.000 claims 1
• 208000002903 Thalassemia Diseases 0.000 claims 1
• 101150079678 hbb1 gene Proteins 0.000 claims 1
• 239000008194 pharmaceutical composition Substances 0.000 claims 1
• 230000008488 polyadenylation Effects 0.000 claims 1
• 208000007056 sickle cell anemia Diseases 0.000 claims 1