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公开(公告)号:US11352605B2
公开(公告)日:2022-06-07
申请号:US16300869
申请日:2017-05-12
Applicant: Erasmus University Medical Center Rotterdam
Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (i PS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.
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公开(公告)号:US10724092B2
公开(公告)日:2020-07-28
申请号:US15315879
申请日:2015-06-10
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Atze Jacobus Bergsma , Erik van der Wal , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus Reuser
IPC: C12Q1/68 , C12Q1/6876 , C12Q1/6883 , C12Q1/6844 , C12Q1/6851 , C12N15/11 , C12N15/113
Abstract: The disclosure provides method and kits for characterizing spliced m RNA isoforms. The disclosure also provides methods of screening for mutations and oligonucleotides that modulate splicing.
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公开(公告)号:US20180360927A1
公开(公告)日:2018-12-20
申请号:US15781809
申请日:2015-12-07
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Erik van der Wal , Atze Jacobus Bergsma , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus Reuser
IPC: A61K38/47 , A61P3/00 , A61P21/00 , C12N15/113 , A61K31/352 , C12N15/11
Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.
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公开(公告)号:US20170306399A1
公开(公告)日:2017-10-26
申请号:US15315879
申请日:2015-06-10
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Atze Jacobus BERGSMA , Erik van der Wal , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus REUSER
IPC: C12Q1/68 , C12N15/113
CPC classification number: C12Q1/6876 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2320/33 , C12N2330/30 , C12N2330/50 , C12N2330/51 , C12Q1/6846 , C12Q1/6851 , C12Q1/6883 , C12Q2600/136 , C12Q2600/156
Abstract: The disclosure provides method and kits for characterizing spliced m RNA isoforms. The disclosure also provides methods of screening for mutations and oligonucleotides that modulate splicing.
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公开(公告)号:US20220380730A1
公开(公告)日:2022-12-01
申请号:US17833506
申请日:2022-06-06
Applicant: Erasmus University Medical Center Rotterdam
Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (iPS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.
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公开(公告)号:US10993995B2
公开(公告)日:2021-05-04
申请号:US15781809
申请日:2015-12-07
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Erik van der Wal , Atze Jacobus Bergsma , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus Reuser
IPC: A61K38/47 , C12N15/11 , C12N15/113 , A61P21/00 , A61P3/00 , A61K31/352
Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.
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公开(公告)号:US20170247704A1
公开(公告)日:2017-08-31
申请号:US15315887
申请日:2015-06-10
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Atze Jacobus BERGSMA , Erik van der Wal , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus REUSER
IPC: C12N15/113
CPC classification number: C12N15/1137 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3233 , C12N2320/33 , C12N2320/34 , C12Y302/0102
Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.
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公开(公告)号:US20210322526A1
公开(公告)日:2021-10-21
申请号:US17306860
申请日:2021-05-03
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Erik van der Wal , Atze Jacobus Bergsma , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus Reuser
IPC: A61K38/47 , C12N15/11 , C12N15/113 , A61P21/00 , A61P3/00 , A61K31/352
Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.
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公开(公告)号:US10308940B2
公开(公告)日:2019-06-04
申请号:US15315887
申请日:2015-06-10
Applicant: Erasmus University Medical Center Rotterdam
Inventor: Atze Jacobus Bergsma , Erik van der Wal , Wilhelmus Wenceslaus Matthias Pijnappel , Antje Tjitske van der Ploeg , Arnoldus Reuser
IPC: C07H21/04 , C12N15/113 , C12N15/11
Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.
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10.
公开(公告)号:US20190161733A1
公开(公告)日:2019-05-30
申请号:US16300869
申请日:2017-05-12
Applicant: Erasmus University Medical Center Rotterdam
Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (i PS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.
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