JP2019531754A5 - - Google Patents

Info

Publication number

JP2019531754A5

JP2019531754A5 JP2019522537A JP2019522537A JP2019531754A5 JP 2019531754 A5 JP2019531754 A5 JP 2019531754A5 JP 2019522537 A JP2019522537 A JP 2019522537A JP 2019522537 A JP2019522537 A JP 2019522537A JP 2019531754 A5 JP2019531754 A5 JP 2019531754A5

Authority

JP

Japan

Prior art keywords

cell

nuclease

cell according

endogenous

endogenous il2rg

Prior art date

2017-10-31

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
• Global Dossier
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• 210000004027 cell Anatomy 0.000 claims description 34
• 101150047851 IL2RG gene Proteins 0.000 claims description 13
• 101710163270 Nuclease Proteins 0.000 claims description 12
• 230000037430 deletion Effects 0.000 claims description 8
• 238000012217 deletion Methods 0.000 claims description 8
• 230000037431 insertion Effects 0.000 claims description 8
• 238000003780 insertion Methods 0.000 claims description 8
• 210000000130 stem cell Anatomy 0.000 claims description 8
• 238000000034 method Methods 0.000 claims description 7
• 238000003776 cleavage reaction Methods 0.000 claims description 6
• 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 6
• 230000007017 scission Effects 0.000 claims description 6
• 239000013603 viral vector Substances 0.000 claims description 6
• 210000001744 T-lymphocyte Anatomy 0.000 claims description 4
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
• 210000004263 induced pluripotent stem cell Anatomy 0.000 claims description 4
• 108091033319 polynucleotide Proteins 0.000 claims description 4
• 239000002157 polynucleotide Substances 0.000 claims description 4
• 102000040430 polynucleotide Human genes 0.000 claims description 4
• 108090000623 proteins and genes Proteins 0.000 claims description 4
• 101001055227 Homo sapiens Cytokine receptor common subunit gamma Proteins 0.000 claims description 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 description 2
• 108091033409 CRISPR Proteins 0.000 claims description 2
• 238000010354 CRISPR gene editing Methods 0.000 claims description 2
• 102100026234 Cytokine receptor common subunit gamma Human genes 0.000 claims description 2
• 230000004568 DNA-binding Effects 0.000 claims description 2
• 241000702421 Dependoparvovirus Species 0.000 claims description 2
• 201000007142 Omenn syndrome Diseases 0.000 claims description 2
• -1 TALE Proteins 0.000 claims description 2
• 108700019146 Transgenes Proteins 0.000 claims description 2
• 208000023940 X-Linked Combined Immunodeficiency disease Diseases 0.000 claims description 2
• 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 2
• 108020004999 messenger RNA Proteins 0.000 claims description 2
• 230000035772 mutation Effects 0.000 claims description 2
• 239000002773 nucleotide Substances 0.000 claims description 2
• 125000003729 nucleotide group Chemical group 0.000 claims description 2
• 229920001184 polypeptide Polymers 0.000 claims description 2
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
• 102000004169 proteins and genes Human genes 0.000 claims description 2
• 208000002491 severe combined immunodeficiency Diseases 0.000 claims description 2
• 239000013598 vector Substances 0.000 claims description 2
• 238000004519 manufacturing process Methods 0.000 description 1