EA035732B1 - Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза - Google Patents
Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза Download PDFInfo
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- EA035732B1 EA035732B1 EA201791986A EA201791986A EA035732B1 EA 035732 B1 EA035732 B1 EA 035732B1 EA 201791986 A EA201791986 A EA 201791986A EA 201791986 A EA201791986 A EA 201791986A EA 035732 B1 EA035732 B1 EA 035732B1
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- exon
- mrna
- aon
- oligonucleotide
- aon1
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Classifications
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- C—CHEMISTRY; METALLURGY
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
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| PCT/EP2016/055360 WO2016142538A1 (en) | 2015-03-11 | 2016-03-11 | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy |
Publications (2)
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| EA201791986A1 EA201791986A1 (ru) | 2018-01-31 |
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| EA201791986A EA035732B1 (ru) | 2015-03-11 | 2016-03-11 | Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза |
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| HK1253335A1 (zh) * | 2015-06-01 | 2019-06-14 | Sarepta Therapeutics, Inc. | 在vii型胶原蛋白中反义诱导外显子的排除 |
| US12227743B2 (en) | 2018-01-25 | 2025-02-18 | Osaka University | Pharmaceutical composition for treatment of diseases associated with upregulated periostin expression or periostin splice variant switching |
| GB201902735D0 (en) | 2019-02-28 | 2019-04-17 | Proqr Therapeutics Ii Bv | Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| JP2023554180A (ja) * | 2020-12-10 | 2023-12-26 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ジストロフィー性表皮水疱症を治療するための物質および方法。 |
| JPWO2024080311A1 (enExample) * | 2022-10-12 | 2024-04-18 | ||
| CN116555349A (zh) * | 2023-01-09 | 2023-08-08 | 中吉智药(南京)生物技术有限公司 | 一种腺相关病毒载体及其构建方法与应用 |
| CN118001404B (zh) * | 2024-02-06 | 2025-02-25 | 四川大学华西医院 | 抑制C17orf67基因表达试剂在制备治疗获得性大疱表皮松解症药物中的用途 |
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| WO2013053819A1 (en) * | 2011-10-11 | 2013-04-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Exon skipping therapy for dystrophic epidermolysis bullosa |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US7101993B1 (en) | 1990-01-11 | 2006-09-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides containing 2′-O-modified purines |
| US5952221A (en) | 1996-03-06 | 1999-09-14 | Avigen, Inc. | Adeno-associated virus vectors comprising a first and second nucleic acid sequence |
| WO1997041208A1 (en) | 1996-04-26 | 1997-11-06 | Case Western Reserve University | Skin regeneration using mesenchymal stem cells |
| JP2002080662A (ja) | 2000-09-08 | 2002-03-19 | Jsr Corp | ゴム組成物 |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| EP2221376B1 (en) | 2001-06-21 | 2012-11-21 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| AU2002366799A1 (en) | 2001-12-19 | 2003-07-09 | Alwyn Company, Inc. | Allantoin-containing preparations for administration as gels and aerosols |
| US20040096833A1 (en) | 2002-11-16 | 2004-05-20 | Isis Pharmaceuticals Inc. | Modulation of FBP-interacting repressor expression |
| US20040092464A1 (en) | 2002-11-11 | 2004-05-13 | Isis Pharmaceuticals Inc. | Modulation of mitogen-activated protein kinase kinase kinase 11 expression |
| DE10238298A1 (de) | 2002-08-21 | 2004-03-04 | Beiersdorf Ag | Verwendung von Antisense-Oligonucleotiden zur Behandlung von degenerativen Hauterscheinungen |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US8765169B2 (en) | 2004-02-13 | 2014-07-01 | Smith & Nephew, Inc. | Wound healing profile |
| WO2005086768A2 (en) | 2004-03-11 | 2005-09-22 | Albert Einstein College Of Medicine Of Yeshiva University | Enhanced production of functional proteins from defective genes |
| HRP20110352T1 (hr) | 2004-06-28 | 2011-06-30 | The University Of Western Australia | Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe |
| GB0505202D0 (en) | 2005-03-14 | 2005-04-20 | Intercytex Ltd | Skin equivalent culture |
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| PL2049664T3 (pl) | 2006-08-11 | 2012-02-29 | Biomarin Tech Bv | Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA |
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| EP2601294B1 (en) | 2010-08-05 | 2018-11-28 | Academisch Ziekenhuis Leiden h.o.d.n. LUMC | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
| US9078911B2 (en) * | 2011-02-08 | 2015-07-14 | The Charlotte-Mecklenburg Hospital Authority | Antisense oligonucleotides |
| US9603949B2 (en) * | 2011-09-01 | 2017-03-28 | University Of Iowa Research Foundation | Oligonucleotide-based probes for detection of bacterial nucleases |
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| KR20150009521A (ko) | 2012-03-07 | 2015-01-26 | 파이브로셀 테크놀로지스, 인코퍼레이티드 | 국소피부제제 및 개인맞춤형 피부치료방법 |
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-
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Patent Citations (1)
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|---|---|---|---|---|
| WO2013053819A1 (en) * | 2011-10-11 | 2013-04-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Exon skipping therapy for dystrophic epidermolysis bullosa |
Non-Patent Citations (5)
| Title |
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| AARTSMA-RUS ANNEMIEKE M.: "Exon skipping : methods and protocols", vol. 867, 1 January 2012, HUMANA PR., New York [u.a.], ISBN: 978-1-61779-766-8, article TURCZYNSKI SANDRINA; TITEUX MATTHIAS; PIRONON NATHALIE; HOVNANIAN ALAIN: "Antisense-mediated exon skipping to reframe transcripts.", pages: 221 - 238, XP008177335, DOI: 10.1007/978-1-61779-767-5_15 * |
| DATABASE EMBL [online] "Sequence 513357 from Patent EP2213738.", XP002758385, retrieved from EBI * |
| DATABASE Geneseq [online] 21 February 2008 (2008-02-21), HOOD L, BECKMANN PM, JOHNSON R, MARELLI M, LI X: "Human organ specific protein related DNA SEQ:60255.", XP002758384, retrieved from EBI * |
| DATABASE Geneseq [online] 27 March 2003 (2003-03-27), TELERMAN A, AMSON R, TUIJNDER M: "Human tumour suppression/reversion-related DNA sequence SeqID1712.", XP002758383, retrieved from EBI * |
| NINGNING DANG, D�D�E F. MURRELL: "Mutation analysis and characterization of COL7A1 mutations in dystrophic epidermolysis bullosa", EXPERIMENTAL DERMATOLOGY, vol. 17, no. 7, 1 July 2008 (2008-07-01), pages 553 - 568, XP055019941, ISSN: 09066705, DOI: 10.1111/j.1600-0625.2008.00723.x * |
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