AU2019317573B2 - Alternative splicing regulation of gene expression and therapeutic methods - Google Patents

Alternative splicing regulation of gene expression and therapeutic methods

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Publication number
AU2019317573B2
AU2019317573B2 AU2019317573A AU2019317573A AU2019317573B2 AU 2019317573 B2 AU2019317573 B2 AU 2019317573B2 AU 2019317573 A AU2019317573 A AU 2019317573A AU 2019317573 A AU2019317573 A AU 2019317573A AU 2019317573 B2 AU2019317573 B2 AU 2019317573B2
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expression
protein
exon
rna
pyridazin
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AU2019317573A
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AU2019317573A1 (en
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Beverly L. Davidson
Amiel Al HUNDLEY
Alejandro Mas Monteys
Paul T. RANUM
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Children's Hospital of Philadelphia
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Children's Hospital of Philadelphia
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
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    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/357—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having two or more oxygen atoms in the same ring, e.g. crown ethers, guanadrel
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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AU2019317573A 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods Active AU2019317573B2 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862715756P 2018-08-07 2018-08-07
US62/715,756 2018-08-07
US201962837701P 2019-04-23 2019-04-23
US62/837,701 2019-04-23
US201962838223P 2019-04-24 2019-04-24
US62/838,223 2019-04-24
US201962872417P 2019-07-10 2019-07-10
US62/872,417 2019-07-10
PCT/US2019/045401 WO2020033473A1 (en) 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods

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AU2019317573A1 AU2019317573A1 (en) 2021-02-25
AU2019317573B2 true AU2019317573B2 (en) 2026-07-16

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US (1) US20210355509A1 (enExample)
EP (1) EP3833357A4 (enExample)
JP (2) JP7615021B2 (enExample)
KR (1) KR20210042123A (enExample)
CN (1) CN112805009A (enExample)
AU (1) AU2019317573B2 (enExample)
CA (1) CA3108293A1 (enExample)
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Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7013753B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
JP7013754B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
TW202131920A (zh) * 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法
JP2020151570A (ja) * 2020-06-24 2020-09-24 株式会社三洋物産 遊技機
US20240141384A1 (en) * 2021-02-19 2024-05-02 University Of Florida Research Foundation, Incoporated Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes
JP2021079226A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021079225A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021087836A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
JP2021087833A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
US20250346893A1 (en) * 2021-10-22 2025-11-13 Fred Hutchinson Cancer Center Synthetic introns for targeted gene expression
CA3254228A1 (en) 2022-05-18 2023-11-23 The Children's Hospital Of Philadelphia COMPOSITIONS AND METHODS FOR REGULATING INDUCIBLE ALTERNATIVE SPLICING OF GENE EXPRESSION
KR20250039324A (ko) * 2022-05-24 2025-03-20 케이트 테라퓨틱스, 인크. Xlmtm 치료를 위한 조성물
KR102470669B1 (ko) 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
JP7556434B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556433B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556432B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
WO2024060205A1 (zh) * 2022-09-23 2024-03-28 北京基驭医疗科技有限公司 包含基于小分子药物的可变剪接调节元件的核酸分子
CN115747217B (zh) * 2022-10-26 2023-11-28 江苏省人民医院（南京医科大学第一附属医院） 长链非编码rna pdxdc1-as1及其应用
EP4701634A1 (en) * 2023-04-28 2026-03-04 Kriya Therapeutics, Inc. Riboswitches for regulating gene expression and therapeutic methods of using the same
WO2025170984A1 (en) * 2024-02-05 2025-08-14 The Children's Hospital Of Philadelphia Cell-type-specific control of gene expression in auditory hair cells
WO2026085222A2 (en) * 2024-10-16 2026-04-23 Rgenta Therapeutics, Inc. Small-molecule regulated alternative splicing

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110086833A1 (en) * 2008-05-27 2011-04-14 Paushkin Sergey V Methods for treating spinal muscular atrophy
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20000036077A (ko) * 1996-09-12 2000-06-26 한스 루돌프 하우스, 헨리테 브룬너, 베아트리체 귄터 셀룰로스 분해 효소를 발현하는 트랜스젠 식물
GB2404382B (en) * 2003-07-28 2008-01-30 Oxitec Ltd Pest control
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
GB2443186A (en) * 2006-10-25 2008-04-30 Oxitec Ltd Expression system for mediating alternative splicing
US20120149757A1 (en) * 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
EP3406252B1 (en) * 2012-02-10 2020-05-13 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014113540A1 (en) * 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110086833A1 (en) * 2008-05-27 2011-04-14 Paushkin Sergey V Methods for treating spinal muscular atrophy
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HAYES GREGORY M ET AL: "Alternative splicing as a novel of means of regulating the expression of therapeutic genes", CANCER GENE THERAPY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 9, no. 2, 1 February 2002 (2002-02-01), pages 133 - 141 *
VOGEL MARC ET AL: "A small, portable RNA device for the control of exon skipping in mammalian cells", NUCLEIC ACIDS RESEARCH, vol. 46, no. 8, 4 May 2018 (2018-05-04), GB, pages e48 - e48 *

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