RU2763319C2 - Реагенты для лечения окулофарингеальной мышечной дистрофии (офмд) и их применение - Google Patents

Реагенты для лечения окулофарингеальной мышечной дистрофии (офмд) и их применение Download PDF

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RU2763319C2
RU2763319C2 RU2019121950A RU2019121950A RU2763319C2 RU 2763319 C2 RU2763319 C2 RU 2763319C2 RU 2019121950 A RU2019121950 A RU 2019121950A RU 2019121950 A RU2019121950 A RU 2019121950A RU 2763319 C2 RU2763319 C2 RU 2763319C2
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sequence
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nucleic acid
lncrnai
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Ванесса СТРИНГЗ-УФОМБАХ
Дэвид СЬЮХИ
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Бенитек АйПи Холдингз Инк.
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RU2019121950A 2016-12-14 2017-12-14 Реагенты для лечения окулофарингеальной мышечной дистрофии (офмд) и их применение RU2763319C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662434312P 2016-12-14 2016-12-14
US62/434,312 2016-12-14
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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RU2019121950A3 RU2019121950A3 (https=) 2021-01-18
RU2019121950A RU2019121950A (ru) 2021-01-18
RU2763319C2 true RU2763319C2 (ru) 2021-12-28

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US (2) US11142765B2 (https=)
EP (1) EP3555294A4 (https=)
JP (2) JP7233149B2 (https=)
KR (2) KR102667867B1 (https=)
CN (2) CN110382698B (https=)
AU (2) AU2017377661B2 (https=)
CA (1) CA3047154A1 (https=)
IL (2) IL267353B2 (https=)
MX (2) MX2019007110A (https=)
RU (1) RU2763319C2 (https=)
WO (1) WO2018107228A1 (https=)
ZA (1) ZA202106827B (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3555294A4 (en) * 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE
CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
US20220098614A1 (en) 2019-02-28 2022-03-31 Benitec IP Holdings Inc. Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD)
CN114901822A (zh) * 2019-12-23 2022-08-12 维萨梅布有限公司 用于同时调节基因表达的组合物和方法
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
WO2008150897A2 (en) * 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
RU2659423C2 (ru) * 2012-02-16 2018-07-02 ЭйТИР ФАРМА, ИНК. ГИСТИДИЛ-тРНК-СИНТЕТАЗЫ ДЛЯ ЛЕЧЕНИЯ АУТОИММУННЫХ И ВОСПАЛИТЕЛЬНЫХ ЗАБОЛЕВАНИЙ

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2345720A3 (en) * 2001-07-12 2012-01-25 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
AU2008209677B2 (en) * 2007-01-31 2013-05-23 Basf Plant Science Gmbh Plants having enhanced yield-related traits and/or increased abiotic stress resistance, and a method for making the same
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
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
CA3020754C (en) * 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
EP3555294A4 (en) 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008150897A2 (en) * 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
RU2659423C2 (ru) * 2012-02-16 2018-07-02 ЭйТИР ФАРМА, ИНК. ГИСТИДИЛ-тРНК-СИНТЕТАЗЫ ДЛЯ ЛЕЧЕНИЯ АУТОИММУННЫХ И ВОСПАЛИТЕЛЬНЫХ ЗАБОЛЕВАНИЙ

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ANVAR, S.Y. et al., "A decline in PABPN1 induces progressive muscle weakness in Oculopharyngeal muscle dystrophy and in muscle again", Aging, vol. 5, no. 6, (20130601), pages 412 - 426. *
M. RIAZ et al. "PABPN1-Dependent mRNA Processing Induces Muscle Wasting". PLoS Genet 12(5): e1006031. *
M. RIAZ et al. "PABPN1-Dependent mRNA Processing Induces Muscle Wasting". PLoS Genet 12(5): e1006031. ANVAR, S.Y. et al., "A decline in PABPN1 induces progressive muscle weakness in Oculopharyngeal muscle dystrophy and in muscle again", Aging, vol. 5, no. 6, (20130601), pages 412 - 426. *

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JP7431359B2 (ja) 2024-02-14
AU2023222827A1 (en) 2023-09-14
KR20230020586A (ko) 2023-02-10
IL267353B2 (en) 2023-12-01
WO2018107228A1 (en) 2018-06-21
JP2023062092A (ja) 2023-05-02
IL304674B1 (en) 2024-07-01
BR112019012312A8 (pt) 2023-02-28
CN118421624A (zh) 2024-08-02
JP2020503014A (ja) 2020-01-30
US20190330629A1 (en) 2019-10-31
RU2019121950A3 (https=) 2021-01-18
MX2024003643A (es) 2024-04-15
AU2017377661A1 (en) 2019-07-18
IL304674A (en) 2023-09-01
KR20190104151A (ko) 2019-09-06
JP7233149B2 (ja) 2023-03-06
CA3047154A1 (en) 2018-06-21
IL304674B2 (en) 2024-11-01
ZA202106827B (en) 2022-07-27
KR102495222B1 (ko) 2023-02-09
BR112019012312A2 (pt) 2020-02-11
KR102667867B1 (ko) 2024-05-29
EP3555294A4 (en) 2020-08-26
IL267353A (en) 2019-08-29
US20220056446A1 (en) 2022-02-24
CN110382698A (zh) 2019-10-25
US11142765B2 (en) 2021-10-12
MX2019007110A (es) 2019-12-05
AU2023222827B2 (en) 2025-02-27
CN110382698B (zh) 2024-05-10
RU2019121950A (ru) 2021-01-18
AU2017377661B2 (en) 2023-07-27
EP3555294A1 (en) 2019-10-23
US11932852B2 (en) 2024-03-19
IL267353B1 (en) 2023-08-01

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