CY1124522T1 - Ενωσεις και μεθοδοι για ρυθμιση της εκφρασης πρωτεϊνης κινασης μυοτονικης δυστροφιας (dmpk) - Google Patents

Ενωσεις και μεθοδοι για ρυθμιση της εκφρασης πρωτεϊνης κινασης μυοτονικης δυστροφιας (dmpk)

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Publication number
CY1124522T1
CY1124522T1 CY20211100834T CY211100834T CY1124522T1 CY 1124522 T1 CY1124522 T1 CY 1124522T1 CY 20211100834 T CY20211100834 T CY 20211100834T CY 211100834 T CY211100834 T CY 211100834T CY 1124522 T1 CY1124522 T1 CY 1124522T1
Authority
CY
Cyprus
Prior art keywords
compounds
methods
dmpk
expression
dampk
Prior art date
Application number
CY20211100834T
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English (en)
Inventor
Sanjay K. Pandey
Robert A. Macleod
Eric E. Swayze
C. Frank Bennett
Original Assignee
Ionis Pharmaceuticals, Inc.
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 Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of CY1124522T1 publication Critical patent/CY1124522T1/el

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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
    • C12N15/1137—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Genetics & Genomics (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
  • Saccharide Compounds (AREA)

Abstract

Παρεχόμενες στο παρόν είναι μέθοδοι, ενώσεις και συνθέσεις για μείωση έκφρασης ενός DMPK mRNA και πρωτεΐνης σε ένα ζώο. Επίσης παρεχόμενες στο παρόν είναι μέθοδοι, ενώσεις και συνθέσεις για κατά προτίμηση μείωση CUGexp DMPK RNA, μείωση μυοτονίας ή μείωση ματισματοπάθειας σε ένα ζώο. Τέτοιες μέθοδοι, ενώσεις και συνθέσεις είναι χρήσιμες για να θεραπεύεται, αποτρέπεται, καθυστερεί ή βελτιώνεται τύπου 1 μυοτονική δυστροφία ή σύμπτωμα αυτής.
CY20211100834T 2013-08-09 2021-09-22 Ενωσεις και μεθοδοι για ρυθμιση της εκφρασης πρωτεϊνης κινασης μυοτονικης δυστροφιας (dmpk) CY1124522T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361864439P 2013-08-09 2013-08-09
US201361889337P 2013-10-10 2013-10-10
PCT/US2014/050481 WO2015021457A2 (en) 2013-08-09 2014-08-11 Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression

Publications (1)

Publication Number Publication Date
CY1124522T1 true CY1124522T1 (el) 2022-07-22

Family

ID=52462058

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20211100834T CY1124522T1 (el) 2013-08-09 2021-09-22 Ενωσεις και μεθοδοι για ρυθμιση της εκφρασης πρωτεϊνης κινασης μυοτονικης δυστροφιας (dmpk)

Country Status (26)

Country Link
US (3) US20160304877A1 (el)
EP (2) EP3995580A3 (el)
JP (4) JP2016530882A (el)
KR (1) KR102589140B1 (el)
CN (1) CN105452464A (el)
AU (2) AU2014306284B2 (el)
BR (1) BR112016002399A2 (el)
CA (1) CA2920776A1 (el)
CL (1) CL2016000301A1 (el)
CY (1) CY1124522T1 (el)
DK (1) DK3030658T3 (el)
ES (1) ES2895099T3 (el)
HR (1) HRP20211365T1 (el)
HU (1) HUE055867T2 (el)
IL (3) IL243917B (el)
LT (1) LT3030658T (el)
MX (2) MX2016001789A (el)
PH (1) PH12016500263A1 (el)
PL (1) PL3030658T3 (el)
PT (1) PT3030658T (el)
RS (1) RS62403B1 (el)
RU (1) RU2690333C2 (el)
SG (1) SG11201600941YA (el)
SI (1) SI3030658T1 (el)
TW (1) TW201536329A (el)
WO (1) WO2015021457A2 (el)

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