JP2019506166A5 - - Google Patents

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Publication number

JP2019506166A5

JP2019506166A5 JP2018539962A JP2018539962A JP2019506166A5 JP 2019506166 A5 JP2019506166 A5 JP 2019506166A5 JP 2018539962 A JP2018539962 A JP 2018539962A JP 2018539962 A JP2018539962 A JP 2018539962A JP 2019506166 A5 JP2019506166 A5 JP 2019506166A5

Authority

JP

Japan

Prior art keywords

seq

nucleotides

nucleic acid

acid molecule

sequence

Prior art date

2017-01-31

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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• 239000002773 nucleotide Substances 0.000 claims description 82
• 125000003729 nucleotide group Chemical group 0.000 claims description 82
• 150000007523 nucleic acids Chemical class 0.000 claims description 51
• 108020004707 nucleic acids Proteins 0.000 claims description 39
• 102000039446 nucleic acids Human genes 0.000 claims description 39
• 102000001690 Factor VIII Human genes 0.000 claims description 24
• 108010054218 Factor VIII Proteins 0.000 claims description 24
• 229960000301 factor viii Drugs 0.000 claims description 24
• 229920001184 polypeptide Polymers 0.000 claims description 24
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 24
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 24
• 108091028043 Nucleic acid sequence Proteins 0.000 claims description 12
• 230000000694 effects Effects 0.000 claims description 12
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 10
• 108020004705 Codon Proteins 0.000 claims description 6
• 210000004027 cell Anatomy 0.000 claims description 6
• 238000000034 method Methods 0.000 claims description 6
• 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
• 108010076504 Protein Sorting Signals Proteins 0.000 claims description 4
• 210000004899 c-terminal region Anatomy 0.000 claims description 4
• 102000009027 Albumins Human genes 0.000 claims description 2
• 108010088751 Albumins Proteins 0.000 claims description 2
• 101100380342 Catharanthus roseus ASO gene Proteins 0.000 claims description 2
• 108700010070 Codon Usage Proteins 0.000 claims description 2
• 102000009786 Immunoglobulin Constant Regions Human genes 0.000 claims description 2
• 108010009817 Immunoglobulin Constant Regions Proteins 0.000 claims description 2
• 241000713666 Lentivirus Species 0.000 claims description 2
• 102000004338 Transferrin Human genes 0.000 claims description 2
• 108090000901 Transferrin Proteins 0.000 claims description 2
• 230000006978 adaptation Effects 0.000 claims description 2
• 150000001413 amino acids Chemical class 0.000 claims description 2
• 238000012258 culturing Methods 0.000 claims description 2
• 230000000368 destabilizing effect Effects 0.000 claims description 2
• 208000031169 hemorrhagic disease Diseases 0.000 claims description 2
• 238000003780 insertion Methods 0.000 claims description 2
• 230000037431 insertion Effects 0.000 claims description 2
• 238000004519 manufacturing process Methods 0.000 claims description 2
• 239000012581 transferrin Substances 0.000 claims description 2
• 208000032843 Hemorrhage Diseases 0.000 description 1
• 208000034158 bleeding Diseases 0.000 description 1
• 230000000740 bleeding effect Effects 0.000 description 1
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1