ES2803651T3 - Emparejamiento de oligonucleótidos con el exón 73 de col7a1 para la terapia de la epidermólisis ampollosa - Google Patents
Emparejamiento de oligonucleótidos con el exón 73 de col7a1 para la terapia de la epidermólisis ampollosa Download PDFInfo
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- ES2803651T3 ES2803651T3 ES16709465T ES16709465T ES2803651T3 ES 2803651 T3 ES2803651 T3 ES 2803651T3 ES 16709465 T ES16709465 T ES 16709465T ES 16709465 T ES16709465 T ES 16709465T ES 2803651 T3 ES2803651 T3 ES 2803651T3
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- col7a1
- oligonucleotide
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| PCT/EP2016/055360 WO2016142538A1 (en) | 2015-03-11 | 2016-03-11 | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy |
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| ES2803651T3 true ES2803651T3 (es) | 2021-01-28 |
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| 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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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
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| US5952221A (en) | 1996-03-06 | 1999-09-14 | Avigen, Inc. | Adeno-associated virus vectors comprising a first and second nucleic acid sequence |
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| 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 |
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| WO2007069813A1 (en) | 2005-12-16 | 2007-06-21 | Modern Cell & Tissue Technologies Inc. | Methods for culturing mesenchymal stem cell and compositions comprising mesenchymal stem cell for reparing skin defects |
| PT2161038E (pt) | 2006-01-26 | 2014-03-10 | Isis Pharmaceuticals Inc | Composições e suas utilizações dirigidas à huntingtina |
| EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| 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 |
| EP2134848A2 (en) | 2007-03-07 | 2009-12-23 | Aarhus Universitet | Pig model for breast cancer, mitochondria related protein folding disorders and/or epidermolysis bullosa simplex |
| US8077297B2 (en) | 2008-06-30 | 2011-12-13 | Nellcor Puritan Bennett Ireland | Methods and systems for discriminating bands in scalograms |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| 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 |
| JP6177243B2 (ja) * | 2011-10-06 | 2017-08-09 | ミラゲン セラピューティクス， インコーポレイテッド | マイクロｒｎａの調節による全身エネルギーホメオスタシスの制御 |
| 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 |
| KR20150009521A (ko) | 2012-03-07 | 2015-01-26 | 파이브로셀 테크놀로지스, 인코퍼레이티드 | 국소피부제제 및 개인맞춤형 피부치료방법 |
| WO2013173637A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| EP3702461A1 (en) | 2013-07-08 | 2020-09-02 | Institut National De La Sante Et De La Recherche Medicale - Inserm | Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof |
| PT3220924T (pt) | 2014-11-17 | 2022-01-12 | Rev Pharma Corp | Medicamento tópico para lesões da pele e mucosas associadas à epidermólise bolhosa |
| 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 |
| SI3307277T1 (sl) | 2015-06-15 | 2021-02-26 | Tirmed Pharma Ab | Enoverižni oligonukleotidi za uporabo pri zdravljenju motenj kože |
| WO2017078526A2 (en) | 2015-11-05 | 2017-05-11 | Rijksuniversiteit Groningen | Antisense oligonucleotide-mediated exon skipping as systemic therapy for recessive dystrophic epidermolysis bullosa (rdeb). |
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Also Published As
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| ES2803651T3 (es) | Emparejamiento de oligonucleótidos con el exón 73 de col7a1 para la terapia de la epidermólisis ampollosa | |
| EA036791B1 (ru) | Олигонуклеотидная терапия врожденного амавроза лебера | |
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