HRP20210227T1 - Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a - Google Patents

Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a Download PDF

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Publication number
HRP20210227T1
HRP20210227T1 HRP20210227TT HRP20210227T HRP20210227T1 HR P20210227 T1 HRP20210227 T1 HR P20210227T1 HR P20210227T T HRP20210227T T HR P20210227TT HR P20210227 T HRP20210227 T HR P20210227T HR P20210227 T1 HRP20210227 T1 HR P20210227T1
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Croatia
Prior art keywords
pharmaceutical preparation
oligonucleotide
expression
preparation according
pharmaceutically acceptable
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HRP20210227TT
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English (en)
Inventor
Veronica COSTA
Maj Hedtjärn
Marius Hoener
Ravi Jagasia
Mads Aaboe JENSEN
Christoph PATSCH
Lykke PEDERSEN
Søren Vestergaard Rasmussen
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F. Hoffmann - La Roche Ag
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Publication of HRP20210227T1 publication Critical patent/HRP20210227T1/hr

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Claims (12)

1. Protusmjerni oligonukleotid, naznačen time, da oligonukleotid predstavlja oligonukleotidni spoj [image] 1. (CMP ID NO: 626_7), gdje velika slova predstavljaju beta-D-oksi LNA nukleozide, mala slova predstavljaju DNA nukleozide, svi LNA C su 5-metil citozin, a sve internukleozidne veze su fosforotioatne internukleozidne veze.
2. Farmaceutski pripravak, naznačen time, da sadrži oligonukleotid prema patentnom zahtjevu 1 i farmaceutski prihvatljiv razrjeđivač, otapalo, nosač, sol i/ili pomoćno sredstvo.
3. Farmaceutski pripravak prema patentnom zahtjevu 2, naznačen time, da je farmaceutski prihvatljivi razrjeđivač fiziološka otopina puferirana fosfatom (PBS).
4. Farmaceutski pripravak prema patentnom zahtjevu 2 ili 3, naznačen time, da je farmaceutski prihvatljiva sol natrijeva sol.
5. Farmaceutski pripravak prema patentnom zahtjevu 2 ili 3, naznačen time, da je farmaceutski prihvatljiva sol kalijeva sol.
6. Farmaceutski pripravak prema bilo kojem od patentnih zahtjeva 2 do 5, naznačen time, da je za primjenu u obliku lijeka.
7. Postupak in vitro za indukciju ekspresije gena UBE3A u ciljnoj stanici u kojoj je suprimirana ekspresija paternalnog gena UBE3A, naznačen time, da navedeni postupak obuhvaća primjenu oligonukleotida prema patentnom zahtjevu 1 ili farmaceutskog pripravka prema bilo kojem od patentnih zahtjeva 2 do 5, u učinkovitoj količini na navedenoj stanici.
8. Postupak prema patentnom zahtjevu 7, naznačen time, da se ekspresija gena UBE3A povećava za najmanje 40% u usporedbi s kontrolom.
9. Postupak prema patentnom zahtjevu 7 ili 8, naznačen time, da se razina transkripta SNHG14 nizvodno od SNORD109B smanjuje za najmanje 30% u usporedbi s kontrolom.
10. Postupak prema bilo kojem od patentnih zahtjeva 7 do 9, naznačen time, da je ciljna stanica živčana stanica.
11. Postupak prema bilo kojem od patentnih zahtjeva 7 do 10, naznačen time, da ekspresija SNORD115 nije značajno promijenjena u usporedbi s kontrolom.
12. Oligonukleotid prema patentnom zahtjevu 1 ili farmaceutski pripravak prema bilo kojem od patentnih zahtjeva 2 do 5, naznačen time, da se koristi u liječenju ili prevenciji Angelmanova sindroma.
HRP20210227TT 2015-11-12 2021-02-09 Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a HRP20210227T1 (hr)

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US20190144824A1 (en) 2019-05-16
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US20200057052A1 (en) 2020-02-20
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JP2019500851A (ja) 2019-01-17
US11852627B2 (en) 2023-12-26
WO2017081250A1 (en) 2017-05-18
CA3004799A1 (en) 2017-05-18
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PE20181157A1 (es) 2018-07-19
CN108350432A (zh) 2018-07-31
CN108291226A (zh) 2018-07-17
ES2852998T3 (es) 2021-09-14
WO2017081254A1 (en) 2017-05-18
US20190154664A1 (en) 2019-05-23
CR20220077A (es) 2023-04-11
AU2023200410A1 (en) 2023-02-23
CL2021002159A1 (es) 2022-03-11
US20240085402A1 (en) 2024-03-14
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DK3374509T3 (da) 2021-02-15
JP7279000B2 (ja) 2023-05-22

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