DK2334794T3 - Modulering af bcl11a til behandling af hæmoglobinopatier - Google Patents

Modulering af bcl11a til behandling af hæmoglobinopatier Download PDF

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DK2334794T3
DK2334794T3 DK09813730.0T DK09813730T DK2334794T3 DK 2334794 T3 DK2334794 T3 DK 2334794T3 DK 09813730 T DK09813730 T DK 09813730T DK 2334794 T3 DK2334794 T3 DK 2334794T3
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bcl11a
globin
expression
cells
cell
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DK09813730.0T
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Stuart H Orkin
Vijay G Sankaran
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Children's Medical Center Corp
Harvard College
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Claims (7)

1. Fremgangsmåde ex vivo eller in vitro til at øge føtale hæmoglobinniveauer i en celle, hvilken fremgangsmåde omfatter trinnene med etablering af kontakt mellem hæmopoietisk progenitorcelle og en effektiv mængde af en sammensætning omfattende en hæmmer af BCL11A, hvorved føtal hæmoglobinekspression øges i cellen, eller dens afkom, i forhold til cellen før etablering af kontakten, hvor hæmmeren af BCL11A er en BCL11 A-specifik nukleinsyre, der hæmmer BCL11 A-ekspression, og hvor den BCLllA-specifikke nukleinsyre er et BCLllA-specifikt RNA-interferensmiddel, en vektor, der koder for et BCLllA-specifikt RNA-interferensmiddel, et antisense-konstrukt eller -molekyle eller et ribozym.
2. Fremgangsmåde ifølge krav 1, hvor RNA-interferensmidlet omfatter én eller flere af nukleotidsekvenseme ifølge SEQ ID NO: 1-6.
3. Sammensætning omfattende en hæmmer af BCL11A til anvendelse i behandling og/eller forebyggelse af en hæmoglobinopati, hvor inhibitoren af BCL11A er en BCL1 lA-specifik nukleinsyre, der hæmmer BCL11 A-ekspression, og hvor den BCL1 lA-specifikke nukleinsyre er et BCL11 A-specifik RNA-interferensmiddel, en vektor, der koder for et BCLllA-specifikt RNA-interferensmiddel, et antisense-konstrukt eller -molekyle eller et ribozym.
4. Sammensætning til anvendelse i behandling og/eller forebyggelse af en hæmoglobinopati ifølge krav 3, hvor hæmoglobinopatien er en β-hæmoglobinopati, eller en seglcellesygdom eller β-thalassæmi.
5. Sammensætning til anvendelse i behandling og/eller forebyggelse af en hæmoglobinopati ifølge krav 3 eller krav 4, hvor anvendelsen omfatter etablering af kontakt mellem en hæmopoietisk progenitorcelle og sammensætningen ex vivo eller in vitro og administration af cellen eller dens afkom til et pattedyr.
6. Sammensætning til anvendelse i behandling og/eller forebyggelse af en hæmoglobinopati ifølge et hvilket som helst af kravene 3 til 5, hvor sammensætningen endvidere omfatter et farmaceutisk acceptabelt bæremiddel eller fortyndingsmiddel.
7. Sammensætning til anvendelse i behandling og/eller forebyggelse af en hæmoglobinopati ifølge et hvilket som helst af kravene 3 til 6, hvor RNA-interferensmidlet omfatter én eller flere af nukleotidsekvenseme ifølge SEQ ID NO: 1-6.
DK09813730.0T 2008-09-15 2009-09-14 Modulering af bcl11a til behandling af hæmoglobinopatier DK2334794T3 (da)

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US9701708P 2008-09-15 2008-09-15
US22257109P 2009-07-02 2009-07-02
PCT/US2009/056770 WO2010030963A2 (en) 2008-09-15 2009-09-14 Modulation of bcl11a for treatment of hemoglobinopathies

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CA (1) CA2737180C (da)
DK (1) DK2334794T3 (da)
ES (2) ES2615829T3 (da)
HK (1) HK1243727B (da)
MX (2) MX345116B (da)
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