JO3558B1 - تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) - Google Patents

تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))

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Publication number
JO3558B1
JO3558B1 JOP/2015/0247A JOP20150247A JO3558B1 JO 3558 B1 JO3558 B1 JO 3558B1 JO P20150247 A JOP20150247 A JO P20150247A JO 3558 B1 JO3558 B1 JO 3558B1
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JO
Jordan
Prior art keywords
oxidase
methods
hao1
inhibition
compositions
Prior art date
Application number
JOP/2015/0247A
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English (en)
Inventor
Querbes William
Liebow Abigail
V Erbe David
Fitzgerald Kevin
Bettencourt Brian
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Alnylam Pharmaceuticals Inc
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Publication date
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Publication of JO3558B1 publication Critical patent/JO3558B1/ar

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    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
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    • C12N2310/3515Lipophilic moiety, e.g. cholesterol

Abstract

يتعلق الاختراع الحالي بعوامل RNAi، على سبيل المثال، عوامل RNAi مزدوجة الجديلة، تستهدف جين HAO1، وطرق استخدام عوامل RNAi هذه لتثبيط التعبير الجيني عن HAO1 ، وطرق علاج خاضعين يعانون، على سبيل المثال، من PH1.
JOP/2015/0247A 2014-10-10 2015-10-11 تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) JO3558B1 (ar)

Applications Claiming Priority (3)

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US201462062751P 2014-10-10 2014-10-10
US201562147976P 2015-04-15 2015-04-15
US201562214602P 2015-09-04 2015-09-04

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JOP/2020/0115A JOP20200115A1 (ar) 2014-10-10 2014-10-10 تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
JOP/2015/0247A JO3558B1 (ar) 2014-10-10 2015-10-11 تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))

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