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公开(公告)号：EP4104867A3
公开(公告)日：2023-03-01
申请号：EP22166699.3
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M. , KRAINER, Adrian
IPC分类号： A61K48/00 , C12N15/113 , C12Q1/68
摘要： Provided herein are methods and compositions for increasing the expression of ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B, and for treating a subject in need thereof, e.g., a subject with deficient ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B protein expression.
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公开(公告)号：EP4104867A2
公开(公告)日：2022-12-21
申请号：EP22166699.3
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M. , KRAINER, Adrian
IPC分类号： A61K48/00 , C12N15/113 , C12Q1/68
摘要： Provided herein are methods and compositions for increasing the expression of ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B, and for treating a subject in need thereof, e.g., a subject with deficient ATP1A2, CACNA1A, SETD5, SHANK3, NF2, DNMT1, TCF4, RAI1, PEX1, ARSA, EIF2B5, EIF2B1, EIF2B2, NPC1, ADAR, MFSD8, STXBP1, PRICKLE2, PRRT2, IDUA, or STX1B protein expression.
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公开(公告)号：EP3389672A1
公开(公告)日：2018-10-24
申请号：EP16876543.6
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M. , HALL, Samuel W. , JING, Enxuan
IPC分类号： A61K31/7088 , C12N15/113 , C12Q1/68
CPC分类号： A61K31/7088 , A61K31/712 , A61K31/7125 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/346 , C12N2320/33 , C12N2310/321 , C12N2310/3521
摘要： Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a liver disease.
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公开(公告)号：EP3390635A1
公开(公告)日：2018-10-24
申请号：EP16876615.2
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M.
IPC分类号： C12N15/113 , C12Q1/68
CPC分类号： C12Q1/6883 , A61K31/7088 , C12N15/1135 , C12N2310/11 , C12Q2600/156 , C12Q2600/178
摘要： Provided herein are methods and compositions for increasing the expression of TSC2, and for treating a subject in need thereof, such as a subject with deficient tuberin protein expression or a subject having tuberous sclerosis complex.
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公开(公告)号：EP3389671A1
公开(公告)日：2018-10-24
申请号：EP16876499.1
申请日：2016-12-13
发明人： AZNAREZ, Isabel , NASH, Huw M.
IPC分类号： A61K31/7088 , C12N15/11 , C12N15/113 , C12Q1/68
CPC分类号： A61K31/7088 , C12N15/111 , C12N15/113 , C12N2310/315 , C12N2310/321 , C12N2320/33 , C12Q1/68 , C12Q1/6883 , C12Q2600/156 , C12N2310/3521
摘要： Provided herein are methods and compositions for increasing the expression of JAGl, and for treating a subject in need thereof, e.g., a subject with deficient JAGl protein expression or a subject having Alagille syndrome (ALGS).
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公开(公告)号：EP3933041A1
公开(公告)日：2022-01-05
申请号：EP21167500.4
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M. , KRAINER, Adrian
IPC分类号： C12N15/113 , C12N15/11 , C12Q1/68 , A61K31/712 , A61K31/7125 , A61K31/713 , C12Q1/6883 , A61P43/00
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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公开(公告)号：EP3390666A1
公开(公告)日：2018-10-24
申请号：EP16876548.5
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M.
IPC分类号： C12Q1/68 , C12N15/11 , C12N15/113 , C12N15/52
CPC分类号： C12N15/1138 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2320/33 , C12N2310/3521
摘要： Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a kidney disease.
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8.发明公开
公开(公告)号：EP3390636A1
公开(公告)日：2018-10-24
申请号：EP16876616.0
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M.
IPC分类号： C12N15/113 , C12N15/11 , C12Q1/68
CPC分类号： C12N15/1138 , A61K31/712 , A61K31/7125 , A61K31/713 , C12N2310/11 , C12N2310/315 , C12N2310/346 , C12N2320/33 , C12Q1/68 , C12Q1/6883 , C12Q2600/158 , C12N2310/321 , C12N2310/3521
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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9.发明公开
公开(公告)号：EP3390642A2
公开(公告)日：2018-10-24
申请号：EP16876606.1
申请日：2016-12-14
发明人： AZNAREZ, Isabel , NASH, Huw M.
IPC分类号： C12N15/85 , C12N15/113 , C12Q1/68
CPC分类号： A61K31/713 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/346 , C12N2320/33
摘要： Provided herein are methods and compositions for increasing the expression of PRPF3 or PRPF8, and for treating a subject in need thereof, e.g., a subject with deficient PRPF3 or PRPF8 protein expression or a subject having Retinitis Pigmentosa.
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公开(公告)号：EP3390634A1
公开(公告)日：2018-10-24
申请号：EP16876610.3
申请日：2016-12-14
IPC分类号： C12N15/113 , C12N15/11 , C12Q1/68
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1138 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2320/33 , C12Q1/6883 , C12N2310/3521
摘要： Provided herein are methods and compositions for increasing the expression of ROM1, TEAD1, RDH5, NR2E3, PAX6, CRX, FSCN2, ABCA4, MYOC, TCF4, MFSD8, CTNS, NXNL1, OPTN, RLBP1, RPE65, LRAT, RDH8, RDH12, RGR, CNGA3, ALMS1, PER1 or IDUA, and for treating a subject in need thereof, e.g., a subject with deficient ROM1, TEAD1, RDH5, NR2E3, PAX6, CRX, FSCN2, ABCA4, MYOC, TCF4, MFSD8, CTNS, NXNL1, OPTN, RLBP1, RPE65, LRAT, RDH8, RDH12, RGR, CNGA3, ALMS1, PER1 or IDUA protein expression.
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