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BR112016007751A2 - composições para modulação de expressão de c9orf72 - Google Patents

composições para modulação de expressão de c9orf72

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Publication number
BR112016007751A2
BR112016007751A2 BR112016007751A BR112016007751A BR112016007751A2 BR 112016007751 A2 BR112016007751 A2 BR 112016007751A2 BR 112016007751 A BR112016007751 A BR 112016007751A BR 112016007751 A BR112016007751 A BR 112016007751A BR 112016007751 A2 BR112016007751 A2 BR 112016007751A2
Authority
BR
Brazil
Prior art keywords
c90rf72
methods
compositions
mrna
exon
Prior art date
Application number
BR112016007751A
Other languages
English (en)
Inventor
Rigo Frank
Singh Priyam
M Freier Susan
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.)
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Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of BR112016007751A2 publication Critical patent/BR112016007751A2/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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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Abstract

a presente invenção refere-se aos métodos e composições para a redução da expressão de mrna e proteína c90rf72 em um animal com inibidores específicos c90rf72. também são descritos aqui métodos e composições para a inibição seletiva de um c90rf72 patogênico associado a mrna variante pela administração de um composto antissenso direcionando à região que começa no sítio de partida do éxon 1a para o sítio de partida do éxon 1b de um c90rf72 pré-mrna. esses métodos são úteis para tratar, prevenir ou melhorar doenças neurodegenerativas em indivíduo com necessidade dos mesmos. inibidores específicos, tais c90rf72 incluem compostos an-tissenso.
BR112016007751A 2013-10-11 2014-10-11 composições para modulação de expressão de c9orf72 BR112016007751A2 (pt)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201361890108P 2013-10-11 2013-10-11
US201361891313P 2013-10-15 2013-10-15
US201361919540P 2013-12-20 2013-12-20
US201461927903P 2014-01-15 2014-01-15
US201461980502P 2014-04-16 2014-04-16
PCT/US2014/060194 WO2015054676A2 (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

Publications (1)

Publication Number Publication Date
BR112016007751A2 true BR112016007751A2 (pt) 2017-09-12

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Application Number Title Priority Date Filing Date
BR112016007751A BR112016007751A2 (pt) 2013-10-11 2014-10-11 composições para modulação de expressão de c9orf72

Country Status (16)

Country Link
US (3) US10221414B2 (pt)
EP (2) EP3055414A4 (pt)
JP (2) JP6679476B2 (pt)
KR (1) KR20160062069A (pt)
CN (2) CN112080502A (pt)
AU (2) AU2014331652B2 (pt)
BR (1) BR112016007751A2 (pt)
CA (1) CA2926408A1 (pt)
CL (3) CL2016000839A1 (pt)
IL (1) IL244759A0 (pt)
MX (2) MX2016004651A (pt)
MY (1) MY192689A (pt)
PH (1) PH12016500603A1 (pt)
RU (1) RU2748426C2 (pt)
SG (2) SG11201602597YA (pt)
WO (1) WO2015054676A2 (pt)

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