PL3291843T3 - Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana - Google Patents

Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana

Info

Publication number
PL3291843T3
PL3291843T3 PL16790226.1T PL16790226T PL3291843T3 PL 3291843 T3 PL3291843 T3 PL 3291843T3 PL 16790226 T PL16790226 T PL 16790226T PL 3291843 T3 PL3291843 T3 PL 3291843T3
Authority
PL
Poland
Prior art keywords
gene
angelman syndrome
therapy approach
gene therapy
modified
Prior art date
Application number
PL16790226.1T
Other languages
English (en)
Inventor
Kevin Ron NASH
Edwin John Weeber
Jennifer Leigh Daily
Original Assignee
University Of South Florida
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 South Florida filed Critical University Of South Florida
Publication of PL3291843T3 publication Critical patent/PL3291843T3/pl

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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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/104—Aminoacyltransferases (2.3.2)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/02—Aminoacyltransferases (2.3.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/02—Acid—amino-acid ligases (peptide synthases)(6.3.2)
    • C12Y603/02019—Ubiquitin-protein ligase (6.3.2.19), i.e. ubiquitin-conjugating enzyme
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Reproductive Health (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Child & Adolescent Psychology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Pregnancy & Childbirth (AREA)
PL16790226.1T 2015-05-07 2016-05-09 Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana PL3291843T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562158269P 2015-05-07 2015-05-07
PCT/US2016/031468 WO2016179584A1 (en) 2015-05-07 2016-05-09 Modified ube3a gene for a gene therapy approach for angelman syndrome

Publications (1)

Publication Number Publication Date
PL3291843T3 true PL3291843T3 (pl) 2023-07-17

Family

ID=57218344

Family Applications (1)

Application Number Title Priority Date Filing Date
PL16790226.1T PL3291843T3 (pl) 2015-05-07 2016-05-09 Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana

Country Status (9)

Country Link
US (2) US11534500B2 (pl)
EP (2) EP4154914B1 (pl)
JP (1) JP6841507B2 (pl)
CN (1) CN107530451A (pl)
CA (1) CA2984629C (pl)
ES (1) ES2947311T3 (pl)
HU (1) HUE062186T2 (pl)
PL (1) PL3291843T3 (pl)
WO (1) WO2016179584A1 (pl)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200138921A1 (en) * 2017-06-23 2020-05-07 The Trustees Of Columbia University In The City Of New York Methods of preventing and treating diseases characterized by synaptic dysfunction and neurodegeneration including alzheimer's disease
EP3645012A4 (en) * 2017-06-28 2021-06-30 University Of South Florida MODIFIED UBE3A GENE FOR A GENE THERAPY APPROACH OF ANGELMAN SYNDROME
CN110237257B (zh) * 2018-03-09 2023-01-03 中国科学院脑科学与智能技术卓越创新中心 Ube3a泛素化PP2A激活因子PTPA在治疗天使综合症和孤独症中的应用
WO2019241624A1 (en) * 2018-06-14 2019-12-19 Ovid Therapeutics Inc. Use of mir-92a or mir-145 in the treatment of angelman syndrome
US20220091137A1 (en) * 2019-01-17 2022-03-24 Hoffmann-La Roche Inc. E3 ubiquitin ligase (ube3a) protein targets
AU2020240136A1 (en) * 2019-03-21 2021-09-30 Ptc Therapeutics, Inc. Vector and method for treating angelman syndrome
MX2021014248A (es) * 2019-05-22 2022-01-06 Univ North Carolina Chapel Hill Genes ube3a y casetes de expresion y su uso.
IT201900008877A1 (it) * 2019-06-13 2020-12-13 Univ Bologna Alma Mater Studiorum Nuovi costrutti per terapia genica
JP2022545184A (ja) * 2019-08-22 2022-10-26 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アンジェルマン症候群の処置のためのｕｂｅ３ａ
WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
WO2022272171A2 (en) * 2021-06-25 2022-12-29 University Of South Florida Secreted ube3a for treatment of neurological disorders

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
AUPP655698A0 (en) * 1998-10-16 1998-11-05 Commonwealth Scientific And Industrial Research Organisation Delivery system for porcine somatotropin
US6706505B1 (en) * 2000-03-08 2004-03-16 Amgen Inc Human E3α ubiquitin ligase family
AU2001257078A1 (en) * 2000-04-18 2001-10-30 Iconix Pharmaceuticals, Inc. Vector and method for targeted replacement and disruption of an integrated dna sequence
AU2001268163A1 (en) 2000-06-01 2001-12-11 Genaissance Pharmaceuticals, Inc. Haplotypes of the ube3a gene
JP2006506044A (ja) * 2002-04-19 2006-02-23 ジョージア テック リサーチ コーポレイション タンパク質分泌ダイナミクスの加速のための組成物及び方法
RU2005138368A (ru) * 2003-06-10 2006-07-27 ЭнЭсДЖЕНЕ А/С (DK) Улучшенная секреция нейбластина
BRPI0418273A (pt) * 2003-12-31 2007-05-02 Sanofi Pasteur Inc imunógenos alvejados
WO2005084714A2 (en) * 2004-03-02 2005-09-15 University Of Florida Research Foundation, Inc. Raav-neprilysin compositions and methods of use
WO2011109398A2 (en) 2010-03-02 2011-09-09 President And Fellows Of Harvard College Methods and compositions for treatment of angelman syndrome and autism spectrum disorders
WO2012064806A2 (en) * 2010-11-11 2012-05-18 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
BR112013021494B1 (pt) * 2011-02-22 2021-09-08 California Institute Of Technology Vetor viral, polinucleotídeo isolado, uso de um vírus adeno-associado (aav) recombinante e método para produzir o mesmo
CA2834255C (en) * 2011-04-26 2021-11-02 Genomix Co., Ltd. Peptide for inducing regeneration of tissue and use thereof
WO2013016279A1 (en) * 2011-07-25 2013-01-31 Beth Israel Deaconess Medical Center, Inc. Animal model of autism
EP3770258B1 (en) 2012-06-25 2024-07-17 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
EP2724721A1 (en) 2012-10-26 2014-04-30 Matentzoglu, Konstantin Composition for use in the treatment of Angelman syndrome and/or autism spectrum disorder, the use of such composition and a method for manufacturing a medicament for the treatment of Angelman syndrome and/or autism spectrum disorder
WO2015127094A1 (en) 2014-02-19 2015-08-27 University Of Florida Research Foundation, Inc. Delivery of nrf2 as therapy for protection against reactive oxygen species
KR102342622B1 (ko) * 2014-06-13 2021-12-22 화이트헤드 인스티튜트 포 바이오메디칼 리서치 아밀로이드 베타 발현 작제물 및 이에 대한 용도

Also Published As

Publication number Publication date
EP3291843A4 (en) 2018-12-05
CN107530451A (zh) 2018-01-02
EP4154914A1 (en) 2023-03-29
US20180104358A1 (en) 2018-04-19
WO2016179584A1 (en) 2016-11-10
US20230277684A1 (en) 2023-09-07
CA2984629C (en) 2024-06-18
US11534500B2 (en) 2022-12-27
CA2984629A1 (en) 2016-11-10
ES2947311T3 (es) 2023-08-04
JP6841507B2 (ja) 2021-03-10
EP3291843A1 (en) 2018-03-14
JP2018518946A (ja) 2018-07-19
EP4154914B1 (en) 2024-08-28
EP3291843B1 (en) 2023-03-22
HUE062186T2 (hu) 2023-09-28

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