DK2576784T3 - Behandling af methionin-sulfoxid-reduktase a (msra) relaterede sygdomme ved hæmning af naturligt antisense-transskript til msra - Google Patents

Behandling af methionin-sulfoxid-reduktase a (msra) relaterede sygdomme ved hæmning af naturligt antisense-transskript til msra Download PDF

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DK2576784T3
DK2576784T3 DK11787293.7T DK11787293T DK2576784T3 DK 2576784 T3 DK2576784 T3 DK 2576784T3 DK 11787293 T DK11787293 T DK 11787293T DK 2576784 T3 DK2576784 T3 DK 2576784T3
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oligonucleotide
msra
nucleic acid
antisense
disease
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Joseph Collard
Sherman Olga Khorkova
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Curna Inc
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Claims (15)

1. Oligonukleotid, der er målrettet et naturligt antisense-transskript til methionin-sulfoxid- reduktase A (MSRA) til anvendelse som en terapeutisk forbindelse, hvor oligonukleotidet øger ekspressionen af methionin-sulfoxid-reduktase A (MSRA), og hvor det naturlige antisense-transskript har nukleinsyresekvensen ifølge SEQ ID NO: 2, eller en variant deraf, der bevarer funktionen af det naturlige antisense-transskript af SEQ ID NO: 2.
2. Oligonukleotid, der er målrettet et naturligt antisense-transskript til methionin-sulfoxid-reduktase A (MSRA) til anvendelse i forebyggelse eller behandling af en methionin-sulfoxid-reduktase A- (MSRA) associeret sygdom eller forstyrrelse, hvor oligonukleotidet øger ekspressionen af methionin-sulfoxid-reduktase A (MSRA), og hvor det naturlige antisense-transskript har nukleinsyresekvensen ifølge SEQ ID NO: 2, eller en variant deraf, der bevarer funktionen af det naturlige antisense-transskript af SEQ ID NO: 2.
3. Anvendelse af et oligonukleotid, der er målrettet et naturligt antisense-transskript til methionin-sulfoxid-reduktase A (MSRA) til fremstilling af et medikament til forebyggelse eller behandling af en methionin-sulfoxid-reduktase A- (MSRA) associeret sygdom eller forstyrrelse, hvor oligonukleotidet øger ekspressionen af methionin-sulfoxid-reduktase A (MSRA), og hvor det naturlige antisense-transskript har nukleinsyresekvensen ifølge SEQ ID NO: 2, eller en variant deraf, der bevarer funktionen af det naturlige antisense-transskript af SEQ ID NO: 2.
4. Anvendelse ifølge krav 3, eller oligonukleotid til anvendelse ifølge krav 2, hvor sygdommen eller forstyrrelsen er udvalgt fra gruppen bestående af en sygdom eller forstyrrelse forbundet med unormal funktion og/eller ekspression af MSRA, cancer, en proliferativ sygdom eller forstyrrelse, en sygdom eller forstyrrelse forbundet med defekte p53-signalvej, en neurologisk sygdom eller forstyrrelse, en nethindesygdom eller forstyrrelse (makuladedegeneration, øjeældning, katarakt, ældning af øje mv.), hudældning, en kardiovaskulær sygdom eller forstyrrelse, en vaskulær sygdom eller forstyrrelse, en aldersrelateret patologi, en infektionssygdom, cellulær oxidativ stress, en nyresygdom eller -forstyrrelse, en nyresygdom eller forstyrrelse, og en sygdom eller forstyrrelse forbundet med neuronal celledød, ældning eller anden tilstand kendetegnet ved uønsket celletab.
5. Fremgangsmåde in vitro til øgning af ekspressionen af methionin-sulfoxid-reduktase A (MSRA) i patientceller eller -væv, der omfatter: etablering af kontakt mellem cellerne og vævene og et oligonukleotid, der er målrettet et naturligt antisense-transskript til methionin-sulfoxid-reduktase A (MSRA); hvorved ekspressionen af methionin-sulfoxid-reduktase A (MSRA) øges, hvor det naturlige antisense-transskript har nukleinsyresekvensen ifølge SEQ ID NO: 2, eller en variant deraf, der bevarer funktionen af det naturlige antisense-transskript af SEQ ID NO: 2.
6. Anvendelse ifølge krav 3 eller 4, eller an oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1, 2 eller 4, eller fremgangsmåde ifølge krav 5, hvor oligonukleotidet er enkeltstrenget.
7. Anvendelse ifølge krav 3 eller 4, eller oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1, 2 eller 4, eller fremgangsmåde ifølge krav 5, hvor oligonukleotidet er en siRNA-forbindelse.
8. Anvendelse ifølge et hvilket som helst af kravene 3, 4, 6 eller 7, eller oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1, 2, 4, 6 eller 7, eller fremgangsmåde ifølge et hvilket som helst af kravene 5 til 7, hvor oligonukleotidet omfatter mindst én af SEQ ID NO: 7 eller 8.
9. Anvendelse ifølge et hvilket som helst af kravene 3, 4 eller 6 til 8, eller oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1, 2, 4 eller 6 til 8, eller fremgangsmåde ifølge et hvilket som helst af kravene 5 til 8, hvor ekspressionen af methionin-sulfoxid-reduktase A (MSRA) øges med mindst 10 %.
10. Anvendelse ifølge et hvilket som helst af kravene 3, 4 eller 6 til 9, eller an oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1, 2, 4 eller 6 til 9, eller fremgangsmåde ifølge et hvilket som helst af kravene 5 til 9, hvor oligonukleotidet endvidere omfatter én eller flere modifikationer, der omfatter: a. mindst én modificeret internukleosidbinding udvalgt fra: et phosphorthioat, alkylphosphonat, phosphordithioat, alkylphosphonthioat, phosphoramidat, carbamat, carbonat, en phosphattriester, et acetamidat, en carboxymethylester og kombinationer deraf; b. mindst ét modificeret nukleotid udvalgt fra: en peptidnukleinsyre (PNA), en låst nukleinsyre (LNA), en arabino-nukleinsyre, en analog, et derivat og kombinationer deraf; eller c. mindst én modificeret sukkerdel udvalgt fra: en 2’-0-methoxyethylmodificeret sukkerdel, en 2’-methoxymodificeret sukkerdel, en 2’-0-alkylmodificeret sukkerdel, en bicyklisk sukkerdel, en 2’-fluordel og kombinationer deraf.
11. Oligonukleotid, der specifikt kan hybridiseres til et naturligt antisense-transskript, der har nukleinsyresekvensen ifølge SEQ ID NO: 2, eller en variant deraf, der bevarer funktionen af det naturlige antisense-transskript af SEQ ID NO: 2.
12. Oligonukleotid ifølge krav 11, hvor oligonukleotidet er enkeltstrenget eller en siRNA-forbindelse.
13. Oligonukleotid ifølge krav 11 eller 12, der omfatter mindst én af SEQ ID NO: 7 eller 8, hvor oligonukleotidet eventuelt endvidere omfatter én eller flere modifikationer, der omfatter: a. mindst én modificeret internukleosidbinding udvalgt fra: et phosphorthioat, alkylphosphonat, phosphordithioat, alkylphosphonthioat, phosphoramidat, carbamat, carbonat, en phosphattriester, et acetamidat, en carboxymethylester og kombinationer deraf; b. mindst ét modificeret nukleotid udvalgt fra: en peptidnukleinsyre (PNA), en låst nukleinsyre (LNA), en arabino-nukleinsyre, en analog, et derivat og kombinationer deraf; eller c. mindst én modificeret sukkerdel udvalgt fra: en 2’-0-methoxyethylmodificeret sukkerdel, en 2’-methoxymodificeret sukkerdel, en 2’-0-alkylmodificeret sukkerdel, en bicyklisk sukkerdel, en 2’-fluordel og kombinationer deraf.
14. Oligonukleotid ifølge et hvilket som helst af kravene 11 til 13, hvor oligonukleotidet er mellem 10 to 30 nukleotider langt.
15. Farmaceutisk sammensætning, der omfatter oligonukleotid ifølge et hvilket som helst af kravene 11 til 14 og en farmaceutisk acceptabel excipiens.
DK11787293.7T 2010-05-26 2011-05-25 Behandling af methionin-sulfoxid-reduktase a (msra) relaterede sygdomme ved hæmning af naturligt antisense-transskript til msra DK2576784T3 (da)

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US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
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