JP2020512815A5 - - Google Patents

Info

Publication number

JP2020512815A5

JP2020512815A5 JP2019554397A JP2019554397A JP2020512815A5 JP 2020512815 A5 JP2020512815 A5 JP 2020512815A5 JP 2019554397 A JP2019554397 A JP 2019554397A JP 2019554397 A JP2019554397 A JP 2019554397A JP 2020512815 A5 JP2020512815 A5 JP 2020512815A5

Authority

JP

Japan

Prior art keywords

sequence

viral vector

seq

promoter

cells

Prior art date

2018-04-02

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
• Global Dossier
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• 239000013603 viral vector Substances 0.000 claims description 33
• 108010069013 Phenylalanine Hydroxylase Proteins 0.000 claims description 26
• 102100038223 Phenylalanine-4-hydroxylase Human genes 0.000 claims description 26
• 239000002245 particle Substances 0.000 claims description 13
• 108091032955 Bacterial small RNA Proteins 0.000 claims description 11
• 210000004027 cell Anatomy 0.000 claims description 10
• 108020004999 messenger RNA Proteins 0.000 claims description 9
• 230000000295 complement effect Effects 0.000 claims description 7
• 210000004185 liver Anatomy 0.000 claims description 6
• 241000713666 Lentivirus Species 0.000 claims description 4
• 201000011252 Phenylketonuria Diseases 0.000 claims description 4
• 108091036066 Three prime untranslated region Proteins 0.000 claims description 4
• 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
• 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 3
• 210000002798 bone marrow cell Anatomy 0.000 claims description 2
• 210000003890 endocrine cell Anatomy 0.000 claims description 2
• 210000002889 endothelial cell Anatomy 0.000 claims description 2
• 210000002919 epithelial cell Anatomy 0.000 claims description 2
• 230000003394 haemopoietic effect Effects 0.000 claims description 2
• 210000003494 hepatocyte Anatomy 0.000 claims description 2
• 210000004698 lymphocyte Anatomy 0.000 claims description 2
• 210000000663 muscle cell Anatomy 0.000 claims description 2
• 210000004412 neuroendocrine cell Anatomy 0.000 claims description 2
• 210000002569 neuron Anatomy 0.000 claims description 2
• 238000004806 packaging method and process Methods 0.000 claims description 2
• 210000004738 parenchymal cell Anatomy 0.000 claims description 2
• 239000002243 precursor Substances 0.000 claims description 2
• 210000004291 uterus Anatomy 0.000 claims description 2
• 108090000790 Enzymes Proteins 0.000 claims 1
• 102000004190 Enzymes Human genes 0.000 claims 1
• 102000004169 proteins and genes Human genes 0.000 claims 1
• 108090000623 proteins and genes Proteins 0.000 claims 1
• 238000000034 method Methods 0.000 description 6
• 241000700605 Viruses Species 0.000 description 2
• 239000013598 vector Substances 0.000 description 2
• 101710091045 Envelope protein Proteins 0.000 description 1
• 101710188315 Protein X Proteins 0.000 description 1
• 108091027967 Small hairpin RNA Proteins 0.000 description 1
• 102100021696 Syncytin-1 Human genes 0.000 description 1
• 238000003745 diagnosis Methods 0.000 description 1
• 238000009609 prenatal screening Methods 0.000 description 1
• 239000004055 small Interfering RNA Substances 0.000 description 1