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公开(公告)号：EP2636740B1
公开(公告)日：2016-11-09
申请号：EP13170239.1
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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公开(公告)号：EP2636741B1
公开(公告)日：2016-04-27
申请号：EP13170245.8
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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13.发明授权Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA 有权的方法和手段进行有效SKIPPING外显子45 Duchenne型肌营养不良症，前体mRNA
公开(公告)号：EP2607484B1
公开(公告)日：2016-01-06
申请号：EP13160338.3
申请日：2009-01-13
发明人： de Kimpe, Josephus Johannes , Platenburg, Gerardus Johannes , van Deutekom, Judith Christina Theodora , Aartsma-Rus, Annemieke , van Ommen, Garrit-Jan Boudewijn
IPC分类号： C12N15/113 , A61K31/7105
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3521
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公开(公告)号：EP2594642B1
公开(公告)日：2015-12-30
申请号：EP12198497.5
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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公开(公告)号：EP2801618A1
公开(公告)日：2014-11-12
申请号：EP14176300.3
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP2636741A1
公开(公告)日：2013-09-11
申请号：EP13170245.8
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP1619249B2
公开(公告)日：2020-01-08
申请号：EP05076770.6
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
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公开(公告)号：EP2940139B1
公开(公告)日：2018-07-04
申请号：EP15166264.0
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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公开(公告)号：EP2594641B1
公开(公告)日：2015-12-30
申请号：EP12198485.0
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
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公开(公告)号：EP2594641A1
公开(公告)日：2013-05-22
申请号：EP12198485.0
申请日：2001-09-21
发明人： van Ommen, Garrit-Jan Boudewijn , van Deutekom, Judith Christina Theodora , Den Dunnen, Johannes Theodorus
IPC分类号： C12N15/11 , A61K48/00 , A61K31/7088
CPC分类号： C12N15/11 , A01K2267/03 , A61K9/127 , A61K31/7088 , A61K48/00 , A61K48/005 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/346 , C12N2320/33 , C12N2750/14142 , C12N2310/3521
摘要： The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
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