JP2020519284A5 - - Google Patents

Info

Publication number

JP2020519284A5

JP2020519284A5 JP2019562332A JP2019562332A JP2020519284A5 JP 2020519284 A5 JP2020519284 A5 JP 2020519284A5 JP 2019562332 A JP2019562332 A JP 2019562332A JP 2019562332 A JP2019562332 A JP 2019562332A JP 2020519284 A5 JP2020519284 A5 JP 2020519284A5

Authority

JP

Japan

Prior art keywords

grna

nucleic acid

seq

protein

itr

Prior art date

2018-05-09

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

• 108020005004 Guide RNA Proteins 0.000 claims 30
• 108090000623 proteins and genes Proteins 0.000 claims 23
• 150000007523 nucleic acids Chemical group 0.000 claims 13
• 102000004169 proteins and genes Human genes 0.000 claims 13
• 238000010362 genome editing Methods 0.000 claims 12
• 108020004707 nucleic acids Proteins 0.000 claims 8
• 102000039446 nucleic acids Human genes 0.000 claims 8
• 210000004962 mammalian cell Anatomy 0.000 claims 7
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 5
• 210000004027 cell Anatomy 0.000 claims 5
• 238000000034 method Methods 0.000 claims 5
• 108091033409 CRISPR Proteins 0.000 claims 4
• 108090000565 Capsid Proteins Proteins 0.000 claims 4
• 102100023321 Ceruloplasmin Human genes 0.000 claims 4
• 230000000295 complement effect Effects 0.000 claims 4
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
• 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
• 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
• 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
• 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
• 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
• 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
• 238000010354 CRISPR gene editing Methods 0.000 claims 2
• 241000702421 Dependoparvovirus Species 0.000 claims 2
• 108700019146 Transgenes Proteins 0.000 claims 2
• 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
• 102000043334 C9orf72 Human genes 0.000 claims 1
• 108700030955 C9orf72 Proteins 0.000 claims 1
• 101150014718 C9orf72 gene Proteins 0.000 claims 1
• 108010016626 Dipeptides Proteins 0.000 claims 1
• 241000124008 Mammalia Species 0.000 claims 1
• 230000015572 biosynthetic process Effects 0.000 claims 1
• 230000015556 catabolic process Effects 0.000 claims 1
• 238000006731 degradation reaction Methods 0.000 claims 1
• 238000012217 deletion Methods 0.000 claims 1
• 230000037430 deletion Effects 0.000 claims 1
• 238000003780 insertion Methods 0.000 claims 1
• 230000037431 insertion Effects 0.000 claims 1
• 238000002955 isolation Methods 0.000 claims 1
• 230000001404 mediated effect Effects 0.000 claims 1
• 239000013608 rAAV vector Substances 0.000 claims 1