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Title:
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
Document Type and Number:
Japanese Patent JP2019150053
Kind Code:
A
Abstract:
To provide exon skipping compositions for treating muscular dystrophy.SOLUTION: Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described. An antisense oligomer of 20 to 50 nucleotides in length binding to a selected target in the human dystrophin gene to induce exon skipping includes a sequence of bases specifically hybridizing to an exon 44 target region selected from the group consisting of H44A(-07+15), H44A(-08+15), H44A(-06+15), H44A(-08+17), H44A(-07+17), and H44A(-06+17).SELECTED DRAWING: None

Inventors:
リチャード ケー. ベストウィック
ダイアン エリザベス フランク
フレッド ジョセフ シュネル
Application Number:
JP2019094121A
Publication Date:
September 12, 2019
Filing Date:
May 17, 2019
Export Citation:
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Assignee:
サレプタ セラピューティクス, インコーポレイテッド
International Classes:
C12N15/113; A61K31/80; A61P21/04; A61P43/00; C08G79/04
Attorney, Agent or Firm:
山本 秀策
森下 夏樹