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1.发明公开公开(公告)号：US20240271111A1
公开(公告)日：2024-08-15
申请号：US18424277
申请日：2024-01-26
发明人： Derek Jantz , James Jefferson Smith
CPC分类号： C12N9/22 , A61K9/51 , A61K38/43 , A61P31/20 , C12N15/52 , C12N15/86 , A61K38/00 , C12N2730/10122 , C12N2750/14141
摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.
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公开(公告)号：US20240156919A1
公开(公告)日：2024-05-16
申请号：US18490484
申请日：2023-10-19
CPC分类号： A61K38/465 , A61K38/54 , C12N7/00 , C12N9/22 , C12N2750/14143
摘要： The invention relates to the field of molecular biology and recombinant nucleic acid technology. In particular, the invention relates to a method of treating a patient with Duchenne Muscular Dystrophy comprising the removal of at least one exon from the dystrophin gene using engineered nucleases.
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公开(公告)号：US11866747B2
公开(公告)日：2024-01-09
申请号：US18161560
申请日：2023-01-30
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143 , C12N2750/14145
摘要： Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been having modified or edited.
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4.发明公开公开(公告)号：US20240002823A1
公开(公告)日：2024-01-04
申请号：US18360517
申请日：2023-07-27
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US11753630B2
公开(公告)日：2023-09-12
申请号：US17931896
申请日：2022-09-13
发明人： Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
CPC分类号： C12N9/22 , C12N15/86 , C12N2750/14143
摘要： The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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公开(公告)号：US20230088311A1
公开(公告)日：2023-03-23
申请号：US17812400
申请日：2022-07-13
发明人： Derek Jantz , James Jefferson Smith
IPC分类号： C12Q1/6811 , C12N9/22 , G06Q99/00
摘要： Methods of cleaving double-stranded DNA that can be recognized and cleaved by a rationally-designed, I-CreI-derived meganuclease are provided. Also provided are recombinant nucleic acids, cells, and organisms containing such recombinant nucleic acids, as well as cells and organisms produced using such meganucleases. Also provided are methods of conducting a custom-designed, I-CreI-derived meganuclease business.
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7.发明申请公开(公告)号：US20220333137A1
公开(公告)日：2022-10-20
申请号：US17585694
申请日：2022-01-27
摘要： Methods of inserting genes into defined locations in the chromosomal DNA of cultured mammalian cell lines which are subject to gene amplification are disclosed. In particular, sequences of interest (e.g., genes encoding biotherapeutic proteins) are inserted proximal to selectable genes in amplifiable loci, and the transformed cells are subjected to selection to induce co-amplification of the selectable gene and the sequence of interest. The invention also relates to meganucleases, vectors and engineered cell lines necessary for performing the methods, to cell lines resulting from the application of the methods, and use of the cell lines to produce protein products of interest.
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8.发明申请公开(公告)号：US20220243187A1
公开(公告)日：2022-08-04
申请号：US17665265
申请日：2022-02-04
发明人： Derek Jantz , James Jefferson Smith
摘要： The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.
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公开(公告)号：US11266693B2
公开(公告)日：2022-03-08
申请号：US17016056
申请日：2020-09-09
发明人： Michael G. Nicholson , James Jefferson Smith , Derek Jantz , Victor Bartsevich , Daniel T. MacLeod , Jeyaraj Antony
摘要： Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human T cell receptor alpha constant region gene. The present disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells.
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公开(公告)号：US20220056427A1
公开(公告)日：2022-02-24
申请号：US17479173
申请日：2021-09-20
发明人： James Jefferson Smith , Janel Lape , Victor Bartsevich , Hui Li
摘要： The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and/or efficiency of indel formation, when compared to the first-generation meganuclease HBV 11-12x.26. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or hepatocellular carcinoma.
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