公开(公告)号：EP3507366A1

公开(公告)日：2019-07-10

申请号：EP17771348.4

申请日：2017-08-31

申请人： ProQR Therapeutics II B.V.

发明人： TURUNEN, Janne Juha ,  AALTO, Antti ,  KLEIN, Bart ,  VAN SINT FIET, Lenka ,  BOUDET, Julien Auguste Germain

IPC分类号： C12N15/113 ,  C12N15/10 ,  A61K31/712

摘要： The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA sequence in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a non-complementary nucleotide in a position opposite to the nucleotide to be edited in the target RNA sequence.

公开(公告)号：EP4268832A2

公开(公告)日：2023-11-01

申请号：EP23189053.4

申请日：2019-03-27

申请人： ProQR Therapeutics II B.V. ,  University Of Rochester

发明人： YU, Yi-Tao ,  ADACHI, Hironori ,  ZOYSA, Meemanage De ,  TURUNEN, Janne Juha ,  KLEIN, Bart ,  VAN SINT FIET, Lenka ,  BOUDET, Julien Auguste Germain ,  DA SILVA, Pedro Duarte Morais Fernandes Arantes

IPC分类号： A61K31/713

摘要： The invention relates to nucleic acid molecules for pseudouridylation of a target uridine in a target RNA in a mammalian cell, wherein the nucleic acid molecule comprises a guide region capable of forming a partially double stranded nucleic acid complex with the target RNA comprising the target uridine, wherein the partially double stranded nucleic acid complex is capable of engaging a mammalian pseudouridylation enzyme, wherein the guide region assists in positioning the target uridine in the partially double stranded nucleic acid complex for it to be converted to a pseudouridine by the mammalian pseudouridylation enzyme.

公开(公告)号：EP3712269A1

公开(公告)日：2020-09-23

申请号：EP20169140.9

申请日：2015-12-17

申请人： ProQR Therapeutics II B.V.

发明人： KLEIN, Bart ,  PLATENBURG, Gerardus Johannes

IPC分类号： C12N15/113

摘要： RNA editing is achieved using oligonucleotide constructs comprising a sequence that is sufficiently complementary to the target RNA sequence to bind by nucleobase pairing to said target RNA, and recognized by an RNA editing entity that is naturally present in said eukaryotic cell. The RNA editing entity, such as ADAR, is redirected to a preselected target site, thereby promoting editing of preselected nucleotide residues in a region of the target RNA.

公开(公告)号：EP3775210A2

公开(公告)日：2021-02-17

申请号：EP19716731.5

申请日：2019-03-27

申请人： University of Rochester ,  ProQR Therapeutics II B.V.

发明人： KLEIN, Bart ,  TURUNEN, Janne, Juha ,  VAN SINT FIET, Lenka ,  DA SILVA, Pedro Duarte Morais Fernandes, Arantes ,  BOUDET, Julien Auguste, Germain ,  YU, Yi-Tao ,  ADACHI, Hironori ,  ZOYSA, Meemanage De

IPC分类号： C12N15/113 ,  C12N15/10 ,  A61K31/713 ,  C12N5/10

公开(公告)号：EP3475424A1

公开(公告)日：2019-05-01

申请号：EP17733425.7

申请日：2017-06-22

申请人： ProQR Therapeutics II B.V.

发明人： TURUNEN, Janne Juha ,  DE BRUIJN, Petra Geziena ,  KLEIN, Bart ,  REDIS, Roxana Simona ,  VAN SINT FIET, Lenka

IPC分类号： C12N15/11

公开(公告)号：EP3947679A1

公开(公告)日：2022-02-09

申请号：EP20719950.6

申请日：2020-04-02

申请人： ProQR Therapeutics II B.V.

发明人： TURUNEN, Janne Juha ,  KLEIN, Bart ,  VAN SINT FIET, Lenka ,  AALTO, Antti ,  KEMMEL, Cherie Paige ,  HOOGEBOOM, Tess ,  VAN WISSEN, Lisanne Alieda

IPC分类号： C12N15/113 ,  C12N15/11 ,  A61K31/7125

公开(公告)号：EP3234134B1

公开(公告)日：2020-05-27

申请号：EP15813826.3

申请日：2015-12-17

申请人： ProQR Therapeutics II B.V.

发明人： KLEIN, Bart ,  PLATENBURG, Gerardus Johannes

IPC分类号： C12N15/113

公开(公告)号：EP4268832A3

公开(公告)日：2024-03-06

申请号：EP23189053.4

申请日：2019-03-27

申请人： ProQR Therapeutics II B.V. ,  University Of Rochester

发明人： YU, Yi-Tao ,  ADACHI, Hironori ,  ZOYSA, Meemanage De ,  TURUNEN, Janne Juha ,  KLEIN, Bart ,  VAN SINT FIET, Lenka ,  BOUDET, Julien Auguste Germain ,  DA SILVA, Pedro Duarte Morais Fernandes Arantes

IPC分类号： C12N15/113 ,  C12N15/10 ,  A61K31/713 ,  C12N5/10

摘要： The invention relates to nucleic acid molecules for pseudouridylation of a target uridine in a target RNA in a mammalian cell, wherein the nucleic acid molecule comprises a guide region capable of forming a partially double stranded nucleic acid complex with the target RNA comprising the target uridine, wherein the partially double stranded nucleic acid complex is capable of engaging a mammalian pseudouridylation enzyme, wherein the guide region assists in positioning the target uridine in the partially double stranded nucleic acid complex for it to be converted to a pseudouridine by the mammalian pseudouridylation enzyme.

公开(公告)号：EP3507366B1

公开(公告)日：2020-10-07

申请号：EP17771348.4

申请日：2017-08-31

申请人： ProQR Therapeutics II B.V.

发明人： TURUNEN, Janne Juha ,  AALTO, Antti ,  KLEIN, Bart ,  VAN SINT FIET, Lenka ,  BOUDET, Julien Auguste Germain

IPC分类号： C12N15/113 ,  C12N15/10 ,  A61K31/712

公开(公告)号：EP3234134A1

公开(公告)日：2017-10-25

申请号：EP15813826.3

申请日：2015-12-17

申请人： ProQR Therapeutics II B.V.

发明人： KLEIN, Bart ,  PLATENBURG, Gerard Johannes

IPC分类号： C12N15/113

摘要： RNA editing is achieved using oligonucleotide constructs comprising (i) a targeting portion specific for a target nucleic acid sequence to be edited and (ii) a recruiting portion capable of binding and recruiting a nucleic acid editing entity naturally present in the cell. The nucleic acid editing entity, such as ADAR, is redirected to a preselected target site by means of the targeting portion, thereby promoting editing of preselected nucleotide residues in a region of the target RNA which corresponds to the targeting portion.

摘要翻译： 使用寡核苷酸构建体实现RNA编辑，所述构建体包含（i）对待编辑的靶核酸序列特异性的靶向部分和（ii）能够结合和招募天然存在于细胞中的核酸编辑实体的招募部分。 诸如ADAR的核酸编辑实体借助于靶向部分被重定向至预选的靶位点，从而促进编码与靶向部分对应的靶RNA区域中的预选核苷酸残基。