AU2019344006B2 - Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome - Google Patents

Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

Info

Publication number
AU2019344006B2
AU2019344006B2 AU2019344006A AU2019344006A AU2019344006B2 AU 2019344006 B2 AU2019344006 B2 AU 2019344006B2 AU 2019344006 A AU2019344006 A AU 2019344006A AU 2019344006 A AU2019344006 A AU 2019344006A AU 2019344006 B2 AU2019344006 B2 AU 2019344006B2
Authority
AU
Australia
Prior art keywords
ube3a
polynucleotide
seq
shrna
rna
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2019344006A
Other languages
English (en)
Other versions
AU2019344006A1 (en
Inventor
Stormy CHAMBERLAIN
Noelle GERMAIN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Connecticut
Original Assignee
University of 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 University of Connecticut filed Critical University of Connecticut
Publication of AU2019344006A1 publication Critical patent/AU2019344006A1/en
Application granted granted Critical
Publication of AU2019344006B2 publication Critical patent/AU2019344006B2/en
Priority to AU2026200604A priority Critical patent/AU2026200604A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/128—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes processing or releasing ribozyme
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2740/15042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • 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
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • 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)
AU2019344006A 2018-09-21 2019-09-20 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome Active AU2019344006B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026200604A AU2026200604A1 (en) 2018-09-21 2026-01-28 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2026200604A Division AU2026200604A1 (en) 2018-09-21 2026-01-28 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

Publications (2)

Publication Number Publication Date
AU2019344006A1 AU2019344006A1 (en) 2021-03-25
AU2019344006B2 true AU2019344006B2 (en) 2025-11-27

Family

ID=69887937

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019344006A Active AU2019344006B2 (en) 2018-09-21 2019-09-20 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome
AU2026200604A Pending AU2026200604A1 (en) 2018-09-21 2026-01-28 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2026200604A Pending AU2026200604A1 (en) 2018-09-21 2026-01-28 Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome

Country Status (10)

Country Link
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) IL281563B2 (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
EP3833398A4 (en) 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS
WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for 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
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
KR102986878B1 (ko) 2022-04-27 2026-07-06 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.
JPH10501681A (ja) 1994-02-22 1998-02-17 ダナ−ファーバー キャンサー インスティチュート 核酸送達システムならびにその合成および使用方法
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
JP2000514290A (ja) 1996-07-01 2000-10-31 ローヌ―プーラン・ロレ・エス・アー 組換えアデノウイルスの製造方法
EP0968284B1 (en) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
IL130198A (en) 1996-12-13 2005-09-25 Schering Corp 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
AU707186B2 (en) 1997-07-01 1999-07-01 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
AU779267B2 (en) 1998-12-31 2005-01-13 Centelion S.A.S. Method for separating viral particles
CA2364934C (fr) 1999-02-22 2011-10-18 Transgene S.A. Procede d'obtention d'une preparation virale purifiee
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
WO2004029212A2 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
AU2004221760B2 (en) 2003-03-21 2010-03-18 Roche Innovation Center Copenhagen A/S Short interfering RNA (siRNA) analogues
ES2356154T3 (es) 2003-07-21 2011-04-05 Transgene S.A. Citoquinas multifuncionales.
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
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS 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
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
SG10201604810UA (en) * 2012-02-07 2016-08-30 Global Bio Therapeutics 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
CN105120901A (zh) 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
WO2014052855A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
WO2015089443A2 (en) * 2013-12-12 2015-06-18 The Brigham And Women's Hospital, Inc. Treating neurodegenerative disease
WO2016086104A1 (en) * 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
HK1258434A1 (zh) * 2015-11-12 2019-11-08 F. Hoffmann-La Roche Ag 来自灵长类物种的标准化神经元细胞测定试验
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN118950038A (zh) 2018-09-07 2024-11-15 科慕埃弗西有限公司 使用含氯烃从氟卤化锑催化剂除去氟
EP3833398A4 (en) 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS

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

Also Published As

Publication number Publication date
MX2021003303A (es) 2021-06-23
JP2022501044A (ja) 2022-01-06
KR102782761B1 (ko) 2025-03-14
KR20210065125A (ko) 2021-06-03
CN113164624B (zh) 2024-09-24
JP7546295B2 (ja) 2024-09-06
WO2020061528A1 (en) 2020-03-26
IL281563A (en) 2021-05-31
AU2019344006A1 (en) 2021-03-25
EP3833398A4 (en) 2021-12-22
CN119592561A (zh) 2025-03-11
AU2026200604A1 (en) 2026-02-19
US20210332368A1 (en) 2021-10-28
IL281563B1 (en) 2026-01-01
IL281563B2 (en) 2026-05-01
EP3833398A1 (en) 2021-06-16
US20250136994A1 (en) 2025-05-01
CN113164624A (zh) 2021-07-23
CA3113648A1 (en) 2020-03-26
US12221609B2 (en) 2025-02-11

Similar Documents

Publication Publication Date Title
US20250136994A1 (en) Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
US20180258424A1 (en) Crispr compositions and methods of using the same for gene therapy
US11932856B2 (en) RNA targeting of mutations via suppressor tRNAs and deaminases
CA3165624A1 (en) Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement
EP4314295A1 (en) Nucleotide editing to reframe dmd transcripts by base editing and prime editing
CA3126886A1 (en) Liver-specific inducible promoters and methods of use thereof
US20260139273A1 (en) Synergistic Nucleic Acid Based Therapeutics and Methods of Use for Treating Genetic Disorders
US20230332150A1 (en) shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
US20230332157A1 (en) shRNA TARGETING UBE3A-ATS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
US20240409936A1 (en) RNAi TARGETING KIF1A MISSENSE MUTATIONS FOR TREATMENT OF KIF1A ASSOCIATED NEUROLOGICAL DISORDER
AU2024374630A1 (en) Methods and compositions for increasing expression of ube3a
US20240409937A1 (en) RNAi TARGETING HNRNPH2 MISSENSE MUTATIONS FOR TREATMENT OF BAIN SYNDROME
US20240409945A1 (en) RNAi TARGETING PPP2R5D MISSENSE MUTATIONS FOR TREATMENT OF JORDAN’S SYNDROME

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)