MX2021008331A - Arn bicatenario y usos del mismo. - Google Patents

Arn bicatenario y usos del mismo.

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Publication number
MX2021008331A
MX2021008331A MX2021008331A MX2021008331A MX2021008331A MX 2021008331 A MX2021008331 A MX 2021008331A MX 2021008331 A MX2021008331 A MX 2021008331A MX 2021008331 A MX2021008331 A MX 2021008331A MX 2021008331 A MX2021008331 A MX 2021008331A
Authority
MX
Mexico
Prior art keywords
mjd
snps
gene
present disclosure
double stranded
Prior art date
Application number
MX2021008331A
Other languages
English (en)
Inventor
Pereira Nobre Rui Jorge Gonçalves
Pereira De Almeida Luís Fernando Morgado
Original Assignee
Univ De Coimbra
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 De Coimbra filed Critical Univ De Coimbra
Publication of MX2021008331A publication Critical patent/MX2021008331A/es

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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 N.A.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (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)

Abstract

La presente divulgación se refiere a un tratamiento no invasivo y específico de alelo, en particular para la enfermedad de Machado-Joseph (MJD); la presente divulgación usa tecnología de silenciamiento de ARN (por ejemplo, interferencia de ARN) contra polimorfismos de un solo nucleótido (SNP) exónicos en el gen de ataxina-3, que codifica para la proteína ataxina-3 mutante de ganancia funcional dominante, dando como resultado de ese modo un tratamiento eficaz para la MJD. Con ese fin, se diseñaron y se sometieron a prueba ARN de silenciamiento génico muy específicos de la diana, cuyas secuencias antisentido son complementarias a SNP que están en desequilibrio de ligamiento con la expansión que provoca la enfermedad; además, esta divulgación también se refiere a un vector viral adenoasociado seleccionado, en particular el serotipo 9 (AAV9) como vector de administración de genes, tras lo cual dichos ARN bicatenarios pueden administrarse al sistema nervioso central (SNC) mediante vías mínimamente invasivas (por ejemplo, administración intravenosa), dado que este serotipo particular atraviesa eficazmente la barrera hematoencefálica (BHE).
MX2021008331A 2019-01-09 2020-01-09 Arn bicatenario y usos del mismo. MX2021008331A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PT11525319 2019-01-09
PCT/IB2020/050141 WO2020144611A1 (en) 2019-01-09 2020-01-09 Double stranded rna and uses thereof

Publications (1)

Publication Number Publication Date
MX2021008331A true MX2021008331A (es) 2021-08-05

Family

ID=69650650

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021008331A MX2021008331A (es) 2019-01-09 2020-01-09 Arn bicatenario y usos del mismo.

Country Status (13)

Country Link
US (1) US20220098592A1 (es)
EP (1) EP3908658A1 (es)
JP (1) JP2022516779A (es)
CN (1) CN113302302A (es)
AU (1) AU2020206617A1 (es)
BR (1) BR112021013109A2 (es)
CA (1) CA3125310A1 (es)
CL (1) CL2021001680A1 (es)
CO (1) CO2021007901A2 (es)
IL (1) IL284717A (es)
MX (1) MX2021008331A (es)
PE (1) PE20211890A1 (es)
WO (1) WO2020144611A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114958850B (zh) * 2021-06-04 2023-12-15 南京大学 一种基因组件、含有此基因组件的递送系统及其应用
WO2024089663A1 (en) 2022-10-27 2024-05-02 Universidade De Coimbra Modified cellular by-product, methods and uses thereof

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
WO2005105995A2 (en) 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP4463608B2 (ja) * 2004-04-19 2010-05-19 独立行政法人科学技術振興機構 変異ＭＪＤ遺伝子の発現を特異的に抑制するｓｉＲＮＡ
US20150315595A1 (en) * 2012-03-12 2015-11-05 Santaris Pharma A/S Compositions and Methods for Modulation of ATXN3 Expression
LT3237618T (lt) * 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
AU2016219396B2 (en) * 2015-02-10 2022-03-17 Genzyme Corporation Variant RNAi
WO2018002886A1 (en) * 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia 3 (sca3) and other related disorders
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease

Also Published As

Publication number Publication date
CL2021001680A1 (es) 2022-02-04
IL284717A (en) 2021-08-31
WO2020144611A1 (en) 2020-07-16
JP2022516779A (ja) 2022-03-02
CO2021007901A2 (es) 2021-07-19
BR112021013109A2 (pt) 2021-10-13
EP3908658A1 (en) 2021-11-17
PE20211890A1 (es) 2021-09-22
CA3125310A1 (en) 2020-07-16
CN113302302A (zh) 2021-08-24
US20220098592A1 (en) 2022-03-31
AU2020206617A1 (en) 2021-08-26
WO2020144611A4 (en) 2020-09-03

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