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MX2022004771A - Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72. - Google Patents

Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72.

Info

Publication number
MX2022004771A
MX2022004771A MX2022004771A MX2022004771A MX2022004771A MX 2022004771 A MX2022004771 A MX 2022004771A MX 2022004771 A MX2022004771 A MX 2022004771A MX 2022004771 A MX2022004771 A MX 2022004771A MX 2022004771 A MX2022004771 A MX 2022004771A
Authority
MX
Mexico
Prior art keywords
vectors
treatment
aav
adeno
c9orf72
Prior art date
Application number
MX2022004771A
Other languages
English (en)
Inventor
Mark Shearman
Steven Pennock
Peixin Zhu
Xijia Wang
Original Assignee
Applied Genetic Tech Corporation
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 Applied Genetic Tech Corporation filed Critical Applied Genetic Tech Corporation
Publication of MX2022004771A publication Critical patent/MX2022004771A/es

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    • 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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • 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
    • 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
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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Abstract

La presente invención proporciona promotores aislados, casetes de expresión transgénica, vectores, kits y métodos para el tratamiento de enfermedades asociadas a C9ORF72, incluyendo ELA y DFT.
MX2022004771A 2019-10-22 2020-10-22 Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72. MX2022004771A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962924351P 2019-10-22 2019-10-22
PCT/US2020/056905 WO2021081236A1 (en) 2019-10-22 2020-10-22 Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases

Publications (1)

Publication Number Publication Date
MX2022004771A true MX2022004771A (es) 2022-10-07

Family

ID=75620858

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004771A MX2022004771A (es) 2019-10-22 2020-10-22 Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72.

Country Status (10)

Country Link
US (2) US20210147873A1 (es)
EP (1) EP4048794A4 (es)
JP (1) JP2023501897A (es)
KR (1) KR20230019063A (es)
CN (1) CN116134134A (es)
AU (1) AU2020370291A1 (es)
CA (1) CA3158518A1 (es)
IL (1) IL292384A (es)
MX (1) MX2022004771A (es)
WO (1) WO2021081236A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3135609A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
TW202308663A (zh) * 2021-06-04 2023-03-01 美商艾拉倫製藥股份有限公司 人類染色體９開讀框７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ劑組成物及其使用方法
WO2023077153A1 (en) * 2021-11-01 2023-05-04 University Of Florida Research Foundation, Incorporated Poly-ga proteins in alzheimer's disease
TW202340467A (zh) * 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
EP4594496A2 (en) * 2022-09-28 2025-08-06 Atalanta Therapeutics, Inc. Compositions and methods for treatment of neurological disorders
CN121057823A (zh) * 2023-04-10 2025-12-02 耶鲁大学 体内修饰神经元以治疗和/或预防肌萎缩性侧索硬化(als)的方法
TW202523337A (zh) * 2023-10-20 2025-06-16 美商星火治療公司 靶向α突觸核蛋白表現之抑制性核酸

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
US9096671B2 (en) * 2011-06-29 2015-08-04 Consejo Superior De Investigaciones Cientificas (Csic) LRP1 as key receptor for the transfer of sterified cholesterol from very-low-density lipoproteins (VLDL) to ischaemic cardiac muscle
EP3202914B2 (en) * 2011-08-31 2023-06-28 The University of Manchester Method for treating a neurodegenerative disease
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
EP3701030A4 (en) * 2017-10-23 2022-04-20 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASES

Also Published As

Publication number Publication date
EP4048794A4 (en) 2024-04-17
WO2021081236A1 (en) 2021-04-29
EP4048794A1 (en) 2022-08-31
KR20230019063A (ko) 2023-02-07
US20210147873A1 (en) 2021-05-20
CN116134134A (zh) 2023-05-16
US20240067984A1 (en) 2024-02-29
JP2023501897A (ja) 2023-01-20
CA3158518A1 (en) 2021-04-29
AU2020370291A1 (en) 2022-05-12
IL292384A (en) 2022-06-01

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