MX2017011599A - Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa. - Google Patents

Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.

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Publication number
MX2017011599A
MX2017011599A MX2017011599A MX2017011599A MX2017011599A MX 2017011599 A MX2017011599 A MX 2017011599A MX 2017011599 A MX2017011599 A MX 2017011599A MX 2017011599 A MX2017011599 A MX 2017011599A MX 2017011599 A MX2017011599 A MX 2017011599A
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MX
Mexico
Prior art keywords
therapy
epidermolysis bullosa
oligonucleotide
oligonucleotides
col7a1 exon
Prior art date
Application number
MX2017011599A
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English (en)
Inventor
Marlene Haisma Elisabeth
Potman Marko
Beumer Wouter
Brinks Vera
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Proqr Therapeutics Ii Bv
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Publication date
Application filed by Proqr Therapeutics Ii Bv filed Critical Proqr Therapeutics Ii Bv
Publication of MX2017011599A publication Critical patent/MX2017011599A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Oligonucleótidos antisentido capaces de prevenir o reducir la inclusión de exón 73 en el mRNA de COL7A humano son caracterizados en varias formas: (a) la secuencia del oligonucleótido incluye cuando mucho dos secuencias de CpG; (b) el oligonucleótido tiene una longitud de no más de 24 nucleótidos; (c) el oligonucleótido es capaz de templar el elemento (unión SRp40/SC35 / ESE) en el exón 73. Estos oligonucleótidos pueden ser útilmente oligoribonucleótidos con enlaces internucleosídicos modificados, por ejemplo, enlaces de fosforotioato.
MX2017011599A 2015-03-11 2016-03-11 Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa. MX2017011599A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides
PCT/EP2016/055360 WO2016142538A1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy

Publications (1)

Publication Number Publication Date
MX2017011599A true MX2017011599A (es) 2018-06-15

Family

ID=52998751

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017011599A MX2017011599A (es) 2015-03-11 2016-03-11 Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.
MX2021009135A MX2021009135A (es) 2015-03-11 2017-09-08 Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.

Family Applications After (1)

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MX2021009135A MX2021009135A (es) 2015-03-11 2017-09-08 Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.

Country Status (20)

Country Link
US (2) US10563198B2 (es)
EP (2) EP3268474B1 (es)
JP (2) JP6929783B2 (es)
KR (1) KR20170120617A (es)
CN (1) CN107567498B (es)
AU (1) AU2016231067B2 (es)
BR (1) BR112017019225A2 (es)
CA (1) CA2978375A1 (es)
DK (1) DK3268474T3 (es)
EA (1) EA035732B1 (es)
ES (1) ES2803651T3 (es)
GB (1) GB201504124D0 (es)
HR (1) HRP20200944T1 (es)
HU (1) HUE050898T2 (es)
IL (1) IL254173B (es)
MX (2) MX2017011599A (es)
PL (1) PL3268474T3 (es)
PT (1) PT3268474T (es)
SI (1) SI3268474T1 (es)
WO (1) WO2016142538A1 (es)

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US20240110181A1 (en) * 2020-12-10 2024-04-04 Institut National de la Santé et de la Recherche Médicale Substances and methods for treating dystrophic epidermolysis bullosa
WO2024080311A1 (ja) * 2022-10-12 2024-04-18 昌和 栗田 遺伝子導入法、遺伝子治療法、及び組織再生法
CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用

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Also Published As

Publication number Publication date
CN107567498B (zh) 2021-09-21
SI3268474T1 (sl) 2020-07-31
US20200399638A1 (en) 2020-12-24
EP3763816A1 (en) 2021-01-13
MX2021009135A (es) 2021-09-10
PT3268474T (pt) 2020-06-17
GB201504124D0 (en) 2015-04-22
ES2803651T3 (es) 2021-01-28
HUE050898T2 (hu) 2021-01-28
EA201791986A1 (ru) 2018-01-31
EP3268474A1 (en) 2018-01-17
US10563198B2 (en) 2020-02-18
NZ736083A (en) 2021-08-27
JP7188810B2 (ja) 2022-12-13
US11352626B2 (en) 2022-06-07
JP6929783B2 (ja) 2021-09-01
AU2016231067A1 (en) 2017-10-26
BR112017019225A2 (pt) 2018-05-08
CN107567498A (zh) 2018-01-09
DK3268474T3 (da) 2020-06-15
HRP20200944T1 (hr) 2020-09-18
JP2018509903A (ja) 2018-04-12
PL3268474T3 (pl) 2020-08-24
CA2978375A1 (en) 2016-09-15
US20180216106A1 (en) 2018-08-02
IL254173B (en) 2022-03-01
KR20170120617A (ko) 2017-10-31
AU2016231067B2 (en) 2022-04-21
IL254173A0 (en) 2017-10-31
EP3268474B1 (en) 2020-05-27
WO2016142538A1 (en) 2016-09-15
JP2022000020A (ja) 2022-01-04
EA035732B1 (ru) 2020-07-31

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