BR112017019971A2 - exclusão de éxon induzida por anti-sentido em miostatina - Google Patents

exclusão de éxon induzida por anti-sentido em miostatina

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Publication number
BR112017019971A2
BR112017019971A2 BR112017019971A BR112017019971A BR112017019971A2 BR 112017019971 A2 BR112017019971 A2 BR 112017019971A2 BR 112017019971 A BR112017019971 A BR 112017019971A BR 112017019971 A BR112017019971 A BR 112017019971A BR 112017019971 A2 BR112017019971 A2 BR 112017019971A2
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BR
Brazil
Prior art keywords
myostatin
antisense
induced exon
exon exclusion
methods
Prior art date
Application number
BR112017019971A
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English (en)
Inventor
Joseph Schnell Frederick
Keith Bestwick Richard
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Sarepta Therapeutics Inc
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Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of BR112017019971A2 publication Critical patent/BR112017019971A2/pt

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    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D265/00—Heterocyclic compounds containing six-membered rings having one nitrogen atom and one oxygen atom as the only ring hetero atoms
    • C07D265/28—1,4-Oxazines; Hydrogenated 1,4-oxazines
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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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
  • Medicinal Preparation (AREA)

Abstract

a presente divulgação refere-se a oligômeros anti-sentido e composições relacionadas, e métodos para diminuir a expressão de miostatina humana funcional e métodos para tratar distrofia muscular e transtornos relacionados, e mais especificamente refere-se a induzir a exclusão de éxon 2 de miostatina e desse modo reduzir os níveis de proteína miostatina.
BR112017019971A 2015-03-18 2016-03-18 exclusão de éxon induzida por anti-sentido em miostatina BR112017019971A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562135048P 2015-03-18 2015-03-18
US201562162571P 2015-05-15 2015-05-15
PCT/US2016/023239 WO2016149659A2 (en) 2015-03-18 2016-03-18 Antisense-induced exon exclusion in myostatin

Publications (1)

Publication Number Publication Date
BR112017019971A2 true BR112017019971A2 (pt) 2018-06-19

Family

ID=56919648

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017019971A BR112017019971A2 (pt) 2015-03-18 2016-03-18 exclusão de éxon induzida por anti-sentido em miostatina

Country Status (6)

Country Link
US (2) US11015200B2 (pt)
EP (1) EP3271372A4 (pt)
JP (2) JP6942054B2 (pt)
BR (1) BR112017019971A2 (pt)
MA (1) MA41795A (pt)
WO (1) WO2016149659A2 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2017047741A1 (ja) * 2015-09-16 2017-03-23 日本新薬株式会社 筋萎縮症治療用アンチセンス核酸
CN117624310A (zh) * 2016-12-30 2024-03-01 奥利通公司 通过肽核酸衍生物的外显子跳跃
WO2020072125A1 (en) * 2018-10-03 2020-04-09 Massachusetts Institute Of Technology Splicing-dependent transcriptional gene silencing or activation

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