KR102782761B1 - 인간의 앤젤만 증후군에서 부계 ube3a 유전자 발현을 복원하기 위한 조성물 및 방법 - Google Patents

인간의 앤젤만 증후군에서 부계 ube3a 유전자 발현을 복원하기 위한 조성물 및 방법 Download PDF

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KR102782761B1
KR102782761B1 KR1020217011023A KR20217011023A KR102782761B1 KR 102782761 B1 KR102782761 B1 KR 102782761B1 KR 1020217011023 A KR1020217011023 A KR 1020217011023A KR 20217011023 A KR20217011023 A KR 20217011023A KR 102782761 B1 KR102782761 B1 KR 102782761B1
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polynucleotide
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nucleotide sequence
shrna
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스토미 챔벌린
노엘 제르망
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유니버시티 오브 코네티컷
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KR1020217011023A 2018-09-21 2019-09-20 인간의 앤젤만 증후군에서 부계 ube3a 유전자 발현을 복원하기 위한 조성물 및 방법 Active KR102782761B1 (ko)

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US201862734435P 2018-09-21 2018-09-21
US62/734,435 2018-09-21
PCT/US2019/052272 WO2020061528A1 (en) 2018-09-21 2019-09-20 Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

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KR102782761B1 true KR102782761B1 (ko) 2025-03-14

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US (2) US12221609B2 (https=)
EP (1) EP3833398A4 (https=)
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CN (2) CN119592561A (https=)
AU (2) AU2019344006B2 (https=)
CA (1) CA3113648A1 (https=)
IL (1) IL281563B1 (https=)
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
EP4142802A4 (en) * 2020-04-28 2024-07-24 The Trustees of The University of Pennsylvania COMPOSITIONS AND USES THEREOF FOR THE TREATMENT OF ANGELMAN SYNDROME
US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
IL315359A (en) * 2022-03-07 2024-11-01 Univ Connecticut shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
KR20230152608A (ko) 2022-04-27 2023-11-03 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150191723A1 (en) * 2012-06-25 2015-07-09 Baylor College Of Medicine Modulation of ube3a-ats expression

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
FR2711523B1 (fr) 1993-10-26 1996-02-16 Transgene Sa Procédé de préparation d'un aérosol viral.
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
US5837520A (en) 1995-03-07 1998-11-17 Canji, Inc. Method of purification of viral vectors
FR2737222B1 (fr) 1995-07-24 1997-10-17 Transgene Sa Nouveaux vecteurs viraux et lignee pour la therapie genique
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
EP0944717A1 (fr) 1996-07-01 1999-09-29 Aventis Pharma S.A. Procede de production d'adenovirus recombinants
ES2278399T3 (es) 1996-11-20 2007-08-01 Introgen Therapeutics, Inc. Metodo mejorado para la produccion y purificacion de vectores adenovirales.
ATE477320T1 (de) 1996-12-13 2010-08-15 Schering Corp Methoden zur virus-reinigung
FR2763958A1 (fr) 1997-05-29 1998-12-04 Transgene Sa Produit de combinaison associant un acide nucleique a une substance desorganisant la matrice extracellulaire pour la therapie genique
CA2242035C (en) 1997-07-01 2004-11-23 Transgene S.A. Compositions useful for transferring therapeutically active substances into a target cell, and their use in gene therapy
JP2002513775A (ja) 1998-05-06 2002-05-14 トランジェーヌ、ソシエテ、アノニム 細胞の中へのポリヌクレオチドのトランスフェクションを改良する治療組成物を調製するためのヌクレアーゼインヒビターまたはインターロイキン−１０（ｉｌ−１０）の使用、および遺伝子治療に有用な組成物
JP3864610B2 (ja) 1998-05-21 2007-01-10 旭硝子株式会社 水分散型撥水撥油剤組成物およびその製造方法
EP0998945A1 (en) 1998-09-30 2000-05-10 Transgene S.A. Use of magnesium (Mg2+) for the enhancement of gene delivery in gene therapy
CA2356373A1 (fr) 1998-12-31 2000-07-13 Aventis Pharma S.A. Methode de separation de particules virales
EP1155120B1 (fr) 1999-02-22 2006-07-05 Transgene S.A. Procede d'obtention d'une preparation virale purifiee
JP2003518511A (ja) 1999-12-28 2003-06-10 トランジェーヌ、ソシエテ、アノニム ポリヌクレオチドを細胞へトランスフェクションするための組成物を調製するためのリチウム（Ｌｉ＋）の使用および遺伝子治療で有用な組成物
CA2500224C (en) 2002-09-25 2015-04-28 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
CA2519860C (en) 2003-03-21 2018-01-16 Santaris Pharma A/S Short interfering rna (sirna) analogues
DK1648931T3 (da) 2003-07-21 2011-03-07 Transgene Sa Multifunktionelle cytokiner
WO2005073378A1 (en) 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
US7361468B2 (en) * 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007124452A2 (en) 2006-04-20 2007-11-01 The Regents Of The University Of California Methods for expressing multiple sirna and shrna from a single vector
US8680064B2 (en) 2008-09-15 2014-03-25 The Board Of Trustees Of The Leland Stanford Junior University ShRNA gene therapy for treatment of ischemic heart disease
WO2011111072A2 (en) 2010-03-10 2011-09-15 Transgene Biotek Ltd. Adeno-associated virus ezh2 shrna (aav ezh2 shrna)therapy for breast cancer
US9163236B2 (en) 2010-12-06 2015-10-20 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Pharmaceutical composition comprising NANOG SHRNA, and method of using NANOG SHRNA to treat cancer
CA2863964C (en) * 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US20130259927A1 (en) 2012-04-02 2013-10-03 Gradalis, Inc. Ewing's Sarcoma Bifunctional shRNA Design
EP2872183B1 (en) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
US10233495B2 (en) 2012-09-27 2019-03-19 The Hospital For Sick Children Methods and compositions for screening and treating developmental disorders
KR20160089528A (ko) * 2013-12-12 2016-07-27 더 브리검 앤드 우먼즈 하스피털, 인크. 신경퇴행성 질환의 치료
WO2016086104A1 (en) * 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
HUE067905T2 (hu) * 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2020051417A2 (en) 2018-09-07 2020-03-12 The Chemours Company Fc, Llc Fluorine removal from antimony fluorohalide catalyst using chlorocarbons
CA3113648A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150191723A1 (en) * 2012-06-25 2015-07-09 Baylor College Of Medicine Modulation of ube3a-ats expression

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