BR112017017178A2 - moduladores específicos de alelo de rodopsina de p23h - Google Patents

moduladores específicos de alelo de rodopsina de p23h

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Publication number
BR112017017178A2
BR112017017178A2 BR112017017178A BR112017017178A BR112017017178A2 BR 112017017178 A2 BR112017017178 A2 BR 112017017178A2 BR 112017017178 A BR112017017178 A BR 112017017178A BR 112017017178 A BR112017017178 A BR 112017017178A BR 112017017178 A2 BR112017017178 A2 BR 112017017178A2
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Brazil
Prior art keywords
rhodopsin
allele
adrp
retinitis
subject
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BR112017017178A
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English (en)
Inventor
L Mccaleb Michael
Singh Priyam
P Seth Punit
F Murray Susan
M Freier Susan
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Ionis Pharmaceuticals Inc
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Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of BR112017017178A2 publication Critical patent/BR112017017178A2/pt

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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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Abstract

as presentes modalidades fornecem métodos, compostos e composições para tratar, prevenir, melhorar ou retardar a progressão da retinite pigmentosa (rp), tal como retinite pigmentosa dominante autossômica (adrp) administrando um inibidor específico de rodopsina p23h para um sujeito. as presentes modalidades apresentadas neste instrumento são direcionadas a compostos e composições úteis para tratar, prevenir, melhor ou retardar a progressão da retinite pigmentosa (rp), como uma retinite pigmentosa dominante autossômica (adrp). em certas modalidades, os inibidores de rodopsina p23h apresentados neste instrumento são compostos antissenso alelo-específicos direcionados a um alelo mutante de p23h que são capazes de inibir, seletivamente, a expressão da proteína mutante da rodopsina de p23h em uma maior extensão do que a proteína de tipo selvagem. em certas modalidades, a administração dos compostos antissenso alelo-específicos em um sujeito com adrp resulta na inibição seletiva de rodopsina de p23h e permite que a proteína normal produzida a partir do alelo de tipo selvagem mantenha sobrevida do bastonete e função no sujeito.
BR112017017178A 2015-02-26 2016-02-26 moduladores específicos de alelo de rodopsina de p23h BR112017017178A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562121286P 2015-02-26 2015-02-26
PCT/US2016/019725 WO2016138353A1 (en) 2015-02-26 2016-02-26 Allele specific modulators of p23h rhodopsin

Publications (1)

Publication Number Publication Date
BR112017017178A2 true BR112017017178A2 (pt) 2018-04-03

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US (3) US10426789B2 (pt)
EP (1) EP3261643B1 (pt)
AU (1) AU2016222546B2 (pt)
BR (1) BR112017017178A2 (pt)
CA (1) CA2977965C (pt)
ES (1) ES2848377T3 (pt)
HK (1) HK1248132A1 (pt)
IL (1) IL253503B (pt)
MX (1) MX2017011010A (pt)
NZ (1) NZ733844A (pt)
WO (1) WO2016138353A1 (pt)

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US20180169131A1 (en) 2018-06-21
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CA2977965A1 (en) 2016-09-01
US11744846B2 (en) 2023-09-05
US10426789B2 (en) 2019-10-01
US20200069722A1 (en) 2020-03-05
AU2016222546A1 (en) 2017-08-03
WO2016138353A1 (en) 2016-09-01
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IL253503B (en) 2021-08-31

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