JPWO2020051561A5 - - Google Patents

Info

Publication number

JPWO2020051561A5

JPWO2020051561A5 JP2021512514A JP2021512514A JPWO2020051561A5 JP WO2020051561 A5 JPWO2020051561 A5 JP WO2020051561A5 JP 2021512514 A JP2021512514 A JP 2021512514A JP 2021512514 A JP2021512514 A JP 2021512514A JP WO2020051561 A5 JPWO2020051561 A5 JP WO2020051561A5

Authority

JP

Japan

Prior art keywords

cas9

terminal fragment

fragment

fused

terminal

Prior art date

2022-09-14

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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• 108091033409 CRISPR Proteins 0.000 claims description 307
• 210000004900 c-terminal fragment Anatomy 0.000 claims description 132
• 108091033319 polynucleotide Proteins 0.000 claims description 126
• 239000002157 polynucleotide Substances 0.000 claims description 126
• 102000040430 polynucleotide Human genes 0.000 claims description 126
• 210000004898 n-terminal fragment Anatomy 0.000 claims description 123
• 108020001507 fusion proteins Proteins 0.000 claims description 73
• 102000037865 fusion proteins Human genes 0.000 claims description 73
• 239000013598 vector Substances 0.000 claims description 52
• 210000004027 cell Anatomy 0.000 claims description 35
• 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 33
• 238000000034 method Methods 0.000 claims description 33
• 239000012634 fragment Substances 0.000 claims description 25
• 101000910035 Streptococcus pyogenes serotype M1 CRISPR-associated endonuclease Cas9/Csn1 Proteins 0.000 claims description 23
• 150000001413 amino acids Chemical class 0.000 claims description 20
• 102000004169 proteins and genes Human genes 0.000 claims description 18
• 108090000623 proteins and genes Proteins 0.000 claims description 18
• 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 17
• 125000003412 L-alanyl group Chemical group [H]N([H])[C@@](C([H])([H])[H])(C(=O)[*])[H] 0.000 claims description 17
• 108020005004 Guide RNA Proteins 0.000 claims description 15
• 230000035772 mutation Effects 0.000 claims description 13
• 210000004899 c-terminal region Anatomy 0.000 claims description 12
• 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims description 10
• 102000055025 Adenosine deaminases Human genes 0.000 claims description 10
• 239000013603 viral vector Substances 0.000 claims description 10
• 125000002842 L-seryl group Chemical group O=C([*])[C@](N([H])[H])([H])C([H])([H])O[H] 0.000 claims description 9
• 125000000769 L-threonyl group Chemical group [H]N([H])[C@]([H])(C(=O)[*])[C@](O[H])(C([H])([H])[H])[H] 0.000 claims description 9
• 125000000729 N-terminal amino-acid group Chemical group 0.000 claims description 8
• 239000000539 dimer Substances 0.000 claims description 8
• 230000000694 effects Effects 0.000 claims description 8
• 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 6
• 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 6
• 241000175212 Herpesvirales Species 0.000 claims description 5
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 5
• 230000001177 retroviral effect Effects 0.000 claims description 5
• 239000004117 Lignosulphonate Substances 0.000 claims description 4
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
• 210000004962 mammalian cell Anatomy 0.000 claims description 4
• 238000006467 substitution reaction Methods 0.000 claims description 4
• 229920001184 polypeptide Polymers 0.000 claims description 3
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 3
• 125000001429 N-terminal alpha-amino-acid group Chemical group 0.000 claims description 2
• 101710163270 Nuclease Proteins 0.000 claims description 2
• 230000017730 intein-mediated protein splicing Effects 0.000 claims description 2
• 239000002773 nucleotide Substances 0.000 claims description 2
• 125000003729 nucleotide group Chemical group 0.000 claims description 2
• 238000000338 in vitro Methods 0.000 claims 3
• 230000001580 bacterial effect Effects 0.000 claims 1
• 241000702421 Dependoparvovirus Species 0.000 description 3
• 241000894006 Bacteria Species 0.000 description 1
• 230000000295 complement effect Effects 0.000 description 1
• 238000012986 modification Methods 0.000 description 1
• 230000004048 modification Effects 0.000 description 1