PL3283500T3 - Kompozycje i sposoby korygowania dystrofii mięśniowej obręczowo-kończynowej typu 2c z użyciem pominięcia egzonu - Google Patents

Kompozycje i sposoby korygowania dystrofii mięśniowej obręczowo-kończynowej typu 2c z użyciem pominięcia egzonu

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Publication number
PL3283500T3
PL3283500T3 PL16777291T PL16777291T PL3283500T3 PL 3283500 T3 PL3283500 T3 PL 3283500T3 PL 16777291 T PL16777291 T PL 16777291T PL 16777291 T PL16777291 T PL 16777291T PL 3283500 T3 PL3283500 T3 PL 3283500T3
Authority
PL
Poland
Prior art keywords
muscle
exon
bypassing
compositions
methods
Prior art date
Application number
PL16777291T
Other languages
English (en)
Inventor
Elizabeth M. MCNALLY
Eugene WYATT
Original Assignee
The University Of Chicago
Northwestern University
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 The University Of Chicago, Northwestern University filed Critical The University Of Chicago
Publication of PL3283500T3 publication Critical patent/PL3283500T3/pl

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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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Footwear And Its Accessory, Manufacturing Method And Apparatuses (AREA)
PL16777291T 2015-04-08 2016-04-07 Kompozycje i sposoby korygowania dystrofii mięśniowej obręczowo-kończynowej typu 2c z użyciem pominięcia egzonu PL3283500T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562144712P 2015-04-08 2015-04-08
PCT/US2016/026477 WO2016164602A1 (en) 2015-04-08 2016-04-07 Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping
EP16777291.2A EP3283500B1 (en) 2015-04-08 2016-04-07 Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping

Publications (1)

Publication Number Publication Date
PL3283500T3 true PL3283500T3 (pl) 2021-05-31

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PL16777291T PL3283500T3 (pl) 2015-04-08 2016-04-07 Kompozycje i sposoby korygowania dystrofii mięśniowej obręczowo-kończynowej typu 2c z użyciem pominięcia egzonu

Country Status (15)

Country Link
US (7) US10273483B2 (pl)
EP (1) EP3283500B1 (pl)
CA (1) CA2981960C (pl)
CY (1) CY1123599T1 (pl)
DK (1) DK3283500T3 (pl)
ES (1) ES2846902T3 (pl)
HR (1) HRP20210139T1 (pl)
HU (1) HUE052604T2 (pl)
LT (1) LT3283500T (pl)
PL (1) PL3283500T3 (pl)
PT (1) PT3283500T (pl)
RS (1) RS61349B1 (pl)
SI (1) SI3283500T1 (pl)
TN (1) TN2017000427A1 (pl)
WO (1) WO2016164602A1 (pl)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2892617T3 (en) * 2012-09-06 2018-09-03 Univ Chicago ANTISENSE POLYNUCLEOTIDES FOR INDUCTION OF EXON SKIPPING AND PROCEDURES FOR TREATING DYSTROPHIES
SI3283500T1 (sl) * 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov

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SI3283500T1 (sl) * 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov

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Publication number Publication date
WO2016164602A1 (en) 2016-10-13
RS61349B1 (sr) 2021-02-26
US10801029B2 (en) 2020-10-13
HRP20210139T1 (hr) 2021-03-19
US20220119820A1 (en) 2022-04-21
US10273483B2 (en) 2019-04-30
HK1250720A1 (zh) 2019-01-11
PT3283500T (pt) 2021-01-28
EP3283500B1 (en) 2020-11-11
BR112017021485A2 (pt) 2018-07-03
US20240301428A1 (en) 2024-09-12
SI3283500T1 (sl) 2020-12-31
DK3283500T3 (en) 2020-11-16
US20260035703A1 (en) 2026-02-05
LT3283500T (lt) 2020-12-10
CY1123599T1 (el) 2022-03-24
US20230167452A1 (en) 2023-06-01
CA2981960A1 (en) 2016-10-13
US20210032631A1 (en) 2021-02-04
TN2017000427A1 (en) 2019-04-12
US20180080030A1 (en) 2018-03-22
HUE052604T2 (hu) 2021-05-28
ES2846902T3 (es) 2021-07-30
EP3283500A1 (en) 2018-02-21
CA2981960C (en) 2023-09-19
EP3283500A4 (en) 2019-01-23
US20190249180A1 (en) 2019-08-15

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