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AR118481A1 - Vector y método para tratar el síndrome de angelman - Google Patents

Vector y método para tratar el síndrome de angelman

Info

Publication number
AR118481A1
AR118481A1 ARP200100816A ARP200100816A AR118481A1 AR 118481 A1 AR118481 A1 AR 118481A1 AR P200100816 A ARP200100816 A AR P200100816A AR P200100816 A ARP200100816 A AR P200100816A AR 118481 A1 AR118481 A1 AR 118481A1
Authority
AR
Argentina
Prior art keywords
vector
angelman syndrome
treat
treat angelman
same
Prior art date
Application number
ARP200100816A
Other languages
English (en)
Inventor
Min Jung Kim
Liangxian Cao
Edwin Weeber
Kevin Nash
Antonio R Arulanandam
Original Assignee
Ptc Therapeutics Inc
Univ 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 Ptc Therapeutics Inc, Univ South Florida filed Critical Ptc Therapeutics Inc
Publication of AR118481A1 publication Critical patent/AR118481A1/es

Links

Classifications

    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C12N15/63—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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Un aspecto descripto en la presente, está relacionado con un virus adeno-asociado recombinante (rAAV), vector y método de uso del mismo para tratar el Síndrome de Angelman. Otro aspecto descripto en la presente es un vector UBE3A rAAV y método de uso del mismo para tratar una deficiencia de UBE3A como por ejemplo el síndrome de Angelman, en humanos.
ARP200100816A 2019-03-21 2020-03-25 Vector y método para tratar el síndrome de angelman AR118481A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962821442P 2019-03-21 2019-03-21

Publications (1)

Publication Number Publication Date
AR118481A1 true AR118481A1 (es) 2021-10-06

Family

ID=72521277

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200100816A AR118481A1 (es) 2019-03-21 2020-03-25 Vector y método para tratar el síndrome de angelman

Country Status (17)

Country Link
US (1) US20220152223A1 (es)
EP (1) EP3941530A4 (es)
JP (1) JP2022525564A (es)
KR (1) KR20210145180A (es)
CN (1) CN114206393A (es)
AR (1) AR118481A1 (es)
AU (1) AU2020240136A1 (es)
BR (1) BR112021018354A2 (es)
CA (1) CA3133455A1 (es)
CL (1) CL2021002427A1 (es)
CO (1) CO2021013967A2 (es)
EA (1) EA202192543A1 (es)
IL (1) IL286476A (es)
MX (1) MX2021011198A (es)
SG (1) SG11202109736RA (es)
TW (1) TW202102672A (es)
WO (1) WO2020191366A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020237130A1 (en) * 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
MX2023006445A (es) * 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
WO2022272171A2 (en) * 2021-06-25 2022-12-29 University Of South Florida Secreted ube3a for treatment of neurological disorders
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001092582A1 (en) * 2000-06-01 2001-12-06 Genaissance Pharmaceuticals, Inc. Haplotypes of the ube3a gene
US20100190656A1 (en) * 2008-08-08 2010-07-29 Integrated Diagnostics, Inc. Breast Cancer Specific Markers and Methods of Use
WO2011133874A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
US9878056B2 (en) * 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9447433B2 (en) * 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CA2927366A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
SI3119437T1 (sl) * 2014-03-21 2020-01-31 Genzyme Corporation Genska terapija za retinitis pigmentoza
US20170348393A1 (en) * 2014-11-06 2017-12-07 Yeda Research And Development Co., Ltd. Treatment of cns inflammatory disorders
CA2984629C (en) * 2015-05-07 2024-06-18 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
FI3411484T3 (fi) * 2016-02-05 2023-11-15 Univ Emory Yksisäikeisen tai itsekomplementaarisen adenoassosioidun viruksen 9 injektio serebrospinaaliseen fluidiin
EP3641794A4 (en) * 2017-06-23 2021-03-24 The Trustees of Columbia University in the City of New York METHODS FOR THE PREVENTION AND TREATMENT OF DISEASES CHARACTERIZED BY SYNAPTIC DYSFUNCTION AND NEURODEGENERENCE, INCLUDING ALZHEIMER'S DISEASE
JP2020528739A (ja) * 2017-06-28 2020-10-01 ユニヴァーシティ オブ サウス フロリダ アンジェルマン症候群の遺伝子治療法のための改変ｕｂｅ３ａ遺伝子
US11845952B2 (en) * 2018-02-07 2023-12-19 Nippon Medical School Foundation Adeno-associated virus vector

Also Published As

Publication number Publication date
EP3941530A1 (en) 2022-01-26
KR20210145180A (ko) 2021-12-01
WO2020191366A1 (en) 2020-09-24
JP2022525564A (ja) 2022-05-17
CL2021002427A1 (es) 2022-07-01
CO2021013967A2 (es) 2022-02-28
SG11202109736RA (en) 2021-10-28
AU2020240136A1 (en) 2021-09-30
EA202192543A1 (ru) 2021-12-27
CA3133455A1 (en) 2020-09-24
MX2021011198A (es) 2022-03-04
EP3941530A4 (en) 2022-12-14
IL286476A (en) 2021-12-01
BR112021018354A2 (pt) 2021-11-23
CN114206393A (zh) 2022-03-18
TW202102672A (zh) 2021-01-16
US20220152223A1 (en) 2022-05-19

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