公开(公告)号：US20220275359A1

公开(公告)日：2022-09-01

申请号：US17631727

申请日：2020-07-31

申请人： Astellas Pharma Inc. ,  Fukuoka University

发明人： Eiji YOSHIMI ,  Yukari MORIYA ,  Mariko MANDA ,  Masatora FUKUDA

IPC分类号： C12N15/10 ,  A61K31/7105 ,  C12N5/10 ,  C12N15/11

摘要： [Problem to be Solved] To provide a guide RNA.
[Solution] A guide RNA for editing a target RNA sequence, comprising an antisense nucleotide sequence complementary to a portion of the target RNA sequence, a short-chain ADAR-recruiting nucleotide sequence, and at least one functional nucleotide sequence.

公开(公告)号：US20210355464A1

公开(公告)日：2021-11-18

申请号：US17320643

申请日：2021-05-14

申请人： ASTELLAS PHARMA INC. ,  MODALIS THERAPEUTICS CORPORATION

发明人： Eiji YOSHIMI ,  Katsuro YOSHIOKA ,  Tetsuya YAMAGATA ,  Yuanbo QIN ,  Iain Robert THOMPSON ,  Nidhi KHANNA

IPC分类号： C12N9/22 ,  C12N15/11 ,  C07K14/47

摘要： Polynucleotides comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcription activator, and (b) a base sequence encoding a guide RNA targeting a continuous region of 18 to 24 nucleotides in length in a region set forth in SEQ ID NO: 104, 105, 135, 141, 153, 167, or 172 in the expression regulatory region of human Utrophin gene are expected to be useful for treating or preventing DUCHENNE muscular dystrophy or BECKER muscular dystrophy.

公开(公告)号：US20230059753A1

公开(公告)日：2023-02-23

申请号：US17783591

申请日：2020-12-08

申请人： Astellas Pharma Inc.

发明人： Eiji YOSHIMI ,  Yukari MORIYA ,  Mariko MANDA ,  Takayasu GOTOH ,  Takatomo ARAI

IPC分类号： C12N15/113 ,  C12N15/10 ,  C12N15/63

摘要： Object: To provide an antisense guide RNA for editing a target RNA by ADAR.
Solution: An antisense guide RNA for editing a target RNA by ADAR, containing at least one functional region and an antisense region that is complementary to a portion of the target RNA and can form a double strand with the target RNA, in which the at least one functional region is linked to the antisense region, and in which the guide RNA does not substantially contain an ADAR-recruiting base sequence.

公开(公告)号：US20220233721A1

公开(公告)日：2022-07-28

申请号：US17614461

申请日：2020-05-27

申请人： Astellas Pharma Inc. ,  Modalis Therapeutics Corporation

发明人： Eiji YOSHIMI ,  Tomoya OE ,  Tetsuya YAMAGATA ,  Keith M. CONNOLLY

IPC分类号： A61K48/00 ,  A61K35/76 ,  A61K38/46 ,  A61K31/7088

摘要： Polynucleotides comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcriptional repressor, and (b) a base sequence encoding a guide RNA targeting a continuous region of 18 to 24 nucleotides in length in a region set forth in SEQ ID NO: 127, SEQ ID NO: 46, SEQ ID NO: 128, SEQ ID NO: 129, SEQ ID NO: 130, SEQ ID NO: 131, SEQ ID NO: 132, SEQ ID NO: 88, SEQ ID NO: 91, SEQ ID NO: 133, SEQ ID NO: 137, SEQ ID NO: 117, or SEQ ID NO: 119 in an expression regulatory region of a human DMPK gene, are expected to be useful for treating muscular dystrophy.