DK3250239T3 - capsid - Google Patents

Info

Publication number

DK3250239T3

DK3250239T3 DK16721917.9T DK16721917T DK3250239T3 DK 3250239 T3 DK3250239 T3 DK 3250239T3 DK 16721917 T DK16721917 T DK 16721917T DK 3250239 T3 DK3250239 T3 DK 3250239T3

Authority

DK

Denmark

Prior art keywords

gly

pro

asn

thr

ser

Prior art date

2015-05-11

Links

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• 210000000234 capsid Anatomy 0.000 title claims description 38
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 29
• 108090000565 Capsid Proteins Proteins 0.000 claims description 29
• 102100023321 Ceruloplasmin Human genes 0.000 claims description 29
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 29
• 239000002773 nucleotide Substances 0.000 claims description 20
• 125000003729 nucleotide group Chemical group 0.000 claims description 20
• 241000958487 Adeno-associated virus 3B Species 0.000 claims description 13
• 241000700605 Viruses Species 0.000 claims description 12
• 241000702421 Dependoparvovirus Species 0.000 claims description 7
• 239000002245 particle Substances 0.000 claims description 7
• 241001465754 Metazoa Species 0.000 claims description 4
• 239000000546 pharmaceutical excipient Substances 0.000 claims description 4
• 238000002560 therapeutic procedure Methods 0.000 claims description 4
• 239000008194 pharmaceutical composition Substances 0.000 claims description 2
• 230000009261 transgenic effect Effects 0.000 claims description 2
• 108090000623 proteins and genes Proteins 0.000 description 55
• 239000013598 vector Substances 0.000 description 55
• 210000004027 cell Anatomy 0.000 description 48
• 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 29
• 238000012546 transfer Methods 0.000 description 26
• 241001634120 Adeno-associated virus - 5 Species 0.000 description 24
• 102000004169 proteins and genes Human genes 0.000 description 24
• 238000010361 transduction Methods 0.000 description 20
• 230000026683 transduction Effects 0.000 description 20
• 239000013607 AAV vector Substances 0.000 description 16
• 108010061238 threonyl-glycine Proteins 0.000 description 15
• 150000007523 nucleic acids Chemical class 0.000 description 14
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• 238000000034 method Methods 0.000 description 13
• 108020004707 nucleic acids Proteins 0.000 description 13
• 102000039446 nucleic acids Human genes 0.000 description 13
• 238000004519 manufacturing process Methods 0.000 description 12
• 108010043121 Green Fluorescent Proteins Proteins 0.000 description 11
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• 239000005090 green fluorescent protein Substances 0.000 description 11
• 241000702423 Adeno-associated virus - 2 Species 0.000 description 9
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 9
• 230000010076 replication Effects 0.000 description 9
• 101100524324 Adeno-associated virus 2 (isolate Srivastava/1982) Rep78 gene Proteins 0.000 description 8
• 108010087924 alanylproline Proteins 0.000 description 8
• KOSRFJWDECSPRO-UHFFFAOYSA-N alpha-L-glutamyl-L-glutamic acid Natural products OC(=O)CCC(N)C(=O)NC(CCC(O)=O)C(O)=O KOSRFJWDECSPRO-UHFFFAOYSA-N 0.000 description 8
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• VPZXBVLAVMBEQI-UHFFFAOYSA-N glycyl-DL-alpha-alanine Natural products OC(=O)C(C)NC(=O)CN VPZXBVLAVMBEQI-UHFFFAOYSA-N 0.000 description 8
• 210000003494 hepatocyte Anatomy 0.000 description 8
• 239000013612 plasmid Substances 0.000 description 8
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• VCSABYLVNWQYQE-SRVKXCTJSA-N Ala-Lys-Lys Chemical compound NCCCC[C@H](NC(=O)[C@@H](N)C)C(=O)N[C@@H](CCCCN)C(O)=O VCSABYLVNWQYQE-SRVKXCTJSA-N 0.000 description 6
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• 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 5
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• YCTIYBUTCKNOTI-UWJYBYFXSA-N Ala-Tyr-Asp Chemical compound C[C@@H](C(=O)N[C@@H](CC1=CC=C(C=C1)O)C(=O)N[C@@H](CC(=O)O)C(=O)O)N YCTIYBUTCKNOTI-UWJYBYFXSA-N 0.000 description 5
• IJPNNYWHXGADJG-GUBZILKMSA-N Arg-Ala-Val Chemical compound [H]N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C)C(=O)N[C@@H](C(C)C)C(O)=O IJPNNYWHXGADJG-GUBZILKMSA-N 0.000 description 5
• JQFJNGVSGOUQDH-XIRDDKMYSA-N Arg-Glu-Trp Chemical compound C1=CC=C2C(C[C@H](NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CCCN=C(N)N)N)C(O)=O)=CNC2=C1 JQFJNGVSGOUQDH-XIRDDKMYSA-N 0.000 description 5
• WIDVAWAQBRAKTI-YUMQZZPRSA-N Asn-Leu-Gly Chemical compound [H]N[C@@H](CC(N)=O)C(=O)N[C@@H](CC(C)C)C(=O)NCC(O)=O WIDVAWAQBRAKTI-YUMQZZPRSA-N 0.000 description 5
• XIDSGDJNUJRUHE-VEVYYDQMSA-N Asn-Thr-Met Chemical compound [H]N[C@@H](CC(N)=O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCSC)C(O)=O XIDSGDJNUJRUHE-VEVYYDQMSA-N 0.000 description 5
• XEDQMTWEYFBOIK-ACZMJKKPSA-N Asp-Ala-Glu Chemical compound [H]N[C@@H](CC(O)=O)C(=O)N[C@@H](C)C(=O)N[C@@H](CCC(O)=O)C(O)=O XEDQMTWEYFBOIK-ACZMJKKPSA-N 0.000 description 5
• UGKZHCBLMLSANF-CIUDSAMLSA-N Asp-Asn-Leu Chemical compound [H]N[C@@H](CC(O)=O)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CC(C)C)C(O)=O UGKZHCBLMLSANF-CIUDSAMLSA-N 0.000 description 5
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• 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 description 5
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