CN110382698B - 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 - Google Patents

用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 Download PDF

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CN110382698B
CN110382698B CN201780086338.5A CN201780086338A CN110382698B CN 110382698 B CN110382698 B CN 110382698B CN 201780086338 A CN201780086338 A CN 201780086338A CN 110382698 B CN110382698 B CN 110382698B
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CN110382698A (zh
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V·斯特林斯-尤弗姆巴
D·苏伊
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Benitec IP Holdings Inc
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CN201780086338.5A 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 Active CN110382698B (zh)

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US201662434312P 2016-12-14 2016-12-14
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PCT/AU2017/051385 WO2018107228A1 (en) 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

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EP (1) EP3555294A4 (cg-RX-API-DMAC10.html)
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102495222B1 (ko) 2016-12-14 2023-02-09 베니텍 아이피 홀딩스 아이엔씨. 안구인두근위축증 (opmd)의 치료제 및 이의 사용
EP3866859A4 (en) * 2018-10-17 2023-08-09 Benitec IP Holdings Inc. METHODS OF TREATMENT FOR OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD)
CA3130221A1 (en) 2019-02-28 2020-09-03 Benitec IP Holdings Inc. Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)
AU2020414441A1 (en) * 2019-12-23 2022-08-18 Versameb Ag Compositions and methods for simultaneously modulating expression of genes
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014077693A1 (en) * 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
CN105143450A (zh) * 2013-01-08 2015-12-09 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR0211111A (pt) * 2001-07-12 2004-06-22 Univ Massachusetts Molécula de ácido nucleico isolada, vetor, célula hospedeira, transgene, precursor de rna engenheirado, animal transgênico não humano, e, método de induzir a interferência de ácido ribonucleico de um gene alvo em uma célula
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
WO2008092935A2 (en) * 2007-01-31 2008-08-07 Basf Plant Science Gmbh Plants having enhanced yield-related traits and/or increased abiotic stress resistance, and a method for making the same
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
EP2814514B1 (en) * 2012-02-16 2017-09-13 Atyr Pharma, Inc. Histidyl-trna synthetases for treating autoimmune and inflammatory diseases
AU2017250017B2 (en) * 2016-04-14 2022-12-22 Benitec IP Holdings Inc. Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof
KR102495222B1 (ko) * 2016-12-14 2023-02-09 베니텍 아이피 홀딩스 아이엔씨. 안구인두근위축증 (opmd)의 치료제 및 이의 사용

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014077693A1 (en) * 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
CN105143450A (zh) * 2013-01-08 2015-12-09 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
A decline in PABPN1 induces progressive muscle weakness in Oculopharyngeal muscle dystrophy and in muscle aging;Seyed Yahya Anvar等;《Aging》;20130630;第5卷(第6期);第413页左栏第1段-第3段，第418页右栏第1段，第423页右栏第4段 *
Adeno-associated virus（AAV）-mediated RNA interference to PABPN1 combined with an potimised resistant transgene for rescue of the muscle specific disease oculopharyngeal muscular dytrophy(OPMD);Houria bachtarzi等;《Abstract of the UK Neuromuscular Translation Research Conference 2014》;20140228;全文 *
Loss of nuclear poly(A)-binding protein 1 causes defects in myogenesis and mRNA biogenesis;Apponi, LH等;《HUMAN MOLECULAR GENETICS》;20100315;第19卷(第6期);全文 *
Progress on gene therapy, cell therapy and pharmacological strategies towards the treatment of Oculopharyngeal Muscular Dystrophy;Pradeep Harish等;《Hum Gene Ther》;20150511;第26卷(第5期);第8页第3段 *

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