JP2017529854A5 - - Google Patents

Info

Publication number

JP2017529854A5

JP2017529854A5 JP2017516515A JP2017516515A JP2017529854A5 JP 2017529854 A5 JP2017529854 A5 JP 2017529854A5 JP 2017516515 A JP2017516515 A JP 2017516515A JP 2017516515 A JP2017516515 A JP 2017516515A JP 2017529854 A5 JP2017529854 A5 JP 2017529854A5

Authority

JP

Japan

Prior art keywords

aav

sequence

replication

nucleotide

seq

Prior art date

2015-09-23

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
• Global Dossier
• Discuss

• 239000002773 nucleotide Substances 0.000 claims 21
• 125000003729 nucleotide group Chemical group 0.000 claims 21
• 238000000034 method Methods 0.000 claims 18
• 230000002950 deficient Effects 0.000 claims 11
• 230000010076 replication Effects 0.000 claims 11
• 108090000565 Capsid Proteins Proteins 0.000 claims 8
• 102100023321 Ceruloplasmin Human genes 0.000 claims 8
• 230000035772 mutation Effects 0.000 claims 8
• 125000003275 alpha amino acid group Chemical group 0.000 claims 6
• 150000001413 amino acids Chemical class 0.000 claims 6
• 210000000415 mammalian chromosome Anatomy 0.000 claims 6
• 210000000130 stem cell Anatomy 0.000 claims 5
• 241000124008 Mammalia Species 0.000 claims 4
• 101710163270 Nuclease Proteins 0.000 claims 4
• 210000000349 chromosome Anatomy 0.000 claims 4
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
• 210000004962 mammalian cell Anatomy 0.000 claims 3
• 108090000623 proteins and genes Proteins 0.000 claims 3
• 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 2
• 241000702421 Dependoparvovirus Species 0.000 claims 2
• 108020005067 RNA Splice Sites Proteins 0.000 claims 2
• 210000000234 capsid Anatomy 0.000 claims 2
• 210000004027 cell Anatomy 0.000 claims 2
• 238000012937 correction Methods 0.000 claims 2
• 238000012217 deletion Methods 0.000 claims 2
• 230000037430 deletion Effects 0.000 claims 2
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
• 239000012634 fragment Substances 0.000 claims 2
• 210000003958 hematopoietic stem cell Anatomy 0.000 claims 2
• 238000010348 incorporation Methods 0.000 claims 2
• 239000012212 insulator Substances 0.000 claims 2
• 208000016718 Chromosome Inversion Diseases 0.000 claims 1
• 108091026890 Coding region Proteins 0.000 claims 1
• 108700024394 Exon Proteins 0.000 claims 1
• 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
• 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
• 108020004684 Internal Ribosome Entry Sites Proteins 0.000 claims 1
• 108091092195 Intron Proteins 0.000 claims 1
• 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
• 206010028980 Neoplasm Diseases 0.000 claims 1
• 108010076504 Protein Sorting Signals Proteins 0.000 claims 1
• 108091036066 Three prime untranslated region Proteins 0.000 claims 1
• 108091023045 Untranslated Region Proteins 0.000 claims 1
• 230000002019 anti-mutation Effects 0.000 claims 1
• 208000035269 cancer or benign tumor Diseases 0.000 claims 1
• 230000002759 chromosomal effect Effects 0.000 claims 1
• 208000037516 chromosome inversion disease Diseases 0.000 claims 1
• 201000010099 disease Diseases 0.000 claims 1
• 208000035475 disorder Diseases 0.000 claims 1
• 210000002889 endothelial cell Anatomy 0.000 claims 1
• 239000003623 enhancer Substances 0.000 claims 1
• 210000004700 fetal blood Anatomy 0.000 claims 1
• 210000002950 fibroblast Anatomy 0.000 claims 1
• 210000003494 hepatocyte Anatomy 0.000 claims 1
• 238000003780 insertion Methods 0.000 claims 1
• 230000037431 insertion Effects 0.000 claims 1
• 210000004698 lymphocyte Anatomy 0.000 claims 1
• 210000004216 mammary stem cell Anatomy 0.000 claims 1
• 210000003098 myoblast Anatomy 0.000 claims 1
• 108091027963 non-coding RNA Proteins 0.000 claims 1
• 102000042567 non-coding RNA Human genes 0.000 claims 1
• 210000005259 peripheral blood Anatomy 0.000 claims 1
• 239000011886 peripheral blood Substances 0.000 claims 1
• 230000002207 retinal effect Effects 0.000 claims 1
• 238000006467 substitution reaction Methods 0.000 claims 1
• 230000026683 transduction Effects 0.000 claims 1
• 238000010361 transduction Methods 0.000 claims 1
• 230000005945 translocation Effects 0.000 claims 1