MX2021003303A - Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. - Google Patents

Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.

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Publication number
MX2021003303A
MX2021003303A MX2021003303A MX2021003303A MX2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A MX 2021003303 A MX2021003303 A MX 2021003303A
Authority
MX
Mexico
Prior art keywords
compositions
human
methods
expression
restore
Prior art date
Application number
MX2021003303A
Other languages
English (en)
Spanish (es)
Inventor
Stormy Chamberlain
Noelle Germain
Original Assignee
Univ Connecticut
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Connecticut filed Critical Univ Connecticut
Publication of MX2021003303A publication Critical patent/MX2021003303A/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
MX2021003303A 2018-09-21 2019-09-20 Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. MX2021003303A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862734435P 2018-09-21 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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MX2021003303A true MX2021003303A (es) 2021-06-23

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US (2) US12221609B2 (enExample)
EP (1) EP3833398A4 (enExample)
JP (1) JP7546295B2 (enExample)
KR (1) KR102782761B1 (enExample)
CN (2) CN119592561A (enExample)
AU (2) AU2019344006B2 (enExample)
CA (1) CA3113648A1 (enExample)
IL (1) IL281563B1 (enExample)
MX (1) MX2021003303A (enExample)
WO (1) WO2020061528A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
JP2023524247A (ja) * 2020-04-28 2023-06-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR20230152608A (ko) 2022-04-27 2023-11-03 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

Family Cites Families (40)

* 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.
EP0745134A1 (en) 1994-02-22 1996-12-04 Danafarber 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
IL127692A0 (en) 1996-07-01 1999-10-28 Rhone Poulenc Rorer Sa Method for producing recombinant adenovirus
WO1998022588A2 (en) 1996-11-20 1998-05-28 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
AU5370598A (en) 1996-12-13 1998-07-03 Schering Corporation Methods for purifying viruses
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
AU752658B2 (en) 1998-05-06 2002-09-26 Association Francaise Contre Les Myopathies Use of a nuclease inhibitor or interleukin-10 (IL-10) for the preparation of a therapeutic composition for improving transfection of a polynucleotide into a cell and compositions useful in gene therapy
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
NZ512504A (en) 1998-12-31 2004-01-30 Aventis Pharma Sa Method for separating viral particles
JP4802366B2 (ja) 1999-02-22 2011-10-26 トランスジェン・ソシエテ・アノニム 精製ウイルス調製物の取得方法
EP1156832A1 (en) 1999-12-28 2001-11-28 Transgene S.A. Use of lithium (li?+ ) for the preparation of a composition for transfection of a polynucleotide into a cell and compositions useful in gene therapy
CA2881743A1 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
ATE443765T1 (de) 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
ATE500267T1 (de) 2003-07-21 2011-03-15 Transgene Sa Multifunktionelle cytokine
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
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
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
WO2010030396A2 (en) 2008-09-15 2010-03-18 Stanford University Novel 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
JP6188728B2 (ja) * 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
US20130259927A1 (en) 2012-04-02 2013-10-03 Gradalis, Inc. Ewing's Sarcoma Bifunctional shRNA Design
WO2014004572A2 (en) * 2012-06-25 2014-01-03 Isis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
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
WO2014052855A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
CA2933554A1 (en) * 2013-12-12 2015-06-18 The Brigham And Women's Hospital, Inc. Treating neurodegenerative disease
US10400243B2 (en) * 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
LT3374509T (lt) * 2015-11-12 2021-03-10 F. Hoffmann-La Roche Ag Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką
US20190224339A1 (en) 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2020051417A2 (en) 2018-09-07 2020-03-12 The Chemours Company Fc, Llc Fluorine removal from antimony fluorohalide catalyst using chlorocarbons
WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

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AU2019344006B2 (en) 2025-11-27
KR20210065125A (ko) 2021-06-03
JP7546295B2 (ja) 2024-09-06
CN113164624B (zh) 2024-09-24
EP3833398A4 (en) 2021-12-22
WO2020061528A1 (en) 2020-03-26
KR102782761B1 (ko) 2025-03-14
US20210332368A1 (en) 2021-10-28
EP3833398A1 (en) 2021-06-16
AU2019344006A1 (en) 2021-03-25
CN119592561A (zh) 2025-03-11
JP2022501044A (ja) 2022-01-06
CA3113648A1 (en) 2020-03-26
US20250136994A1 (en) 2025-05-01
IL281563A (en) 2021-05-31
IL281563B1 (en) 2026-01-01
US12221609B2 (en) 2025-02-11
AU2026200604A1 (en) 2026-02-19
CN113164624A (zh) 2021-07-23

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