AU2018375807B2 - Angelman syndrome antisense treatment - Google Patents

Angelman syndrome antisense treatment Download PDF

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AU2018375807B2
AU2018375807B2 AU2018375807A AU2018375807A AU2018375807B2 AU 2018375807 B2 AU2018375807 B2 AU 2018375807B2 AU 2018375807 A AU2018375807 A AU 2018375807A AU 2018375807 A AU2018375807 A AU 2018375807A AU 2018375807 B2 AU2018375807 B2 AU 2018375807B2
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artificial sequence
synthetic construct
rna
dna
aso
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AU2018375807A1 (en
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Scott Victor DINDOT
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Texas A&M University
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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  • Neurology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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AU2018375807A 2017-12-01 2018-11-30 Angelman syndrome antisense treatment Active AU2018375807B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US62/593,431 2017-12-01
US201862676034P 2018-05-24 2018-05-24
US62/676,034 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (2)

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AU2018375807A1 AU2018375807A1 (en) 2020-05-07
AU2018375807B2 true AU2018375807B2 (en) 2025-04-10

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WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
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BR112020009431A2 (pt) 2020-10-13
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IL274146B1 (en) 2024-07-01
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US20200370046A1 (en) 2020-11-26
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US20250066786A1 (en) 2025-02-27
US11739328B2 (en) 2023-08-29
RU2020121752A (ru) 2021-12-30
EP3717646A4 (en) 2021-12-01
JP2025041632A (ja) 2025-03-26
KR20240161203A (ko) 2024-11-12
AU2018375807A1 (en) 2020-05-07
ES3032315T3 (en) 2025-07-17
KR20200094152A (ko) 2020-08-06
JP2023123517A (ja) 2023-09-05
DK3717646T3 (en) 2025-05-19
IL274146A (en) 2020-06-30
CO2020006361A2 (es) 2020-08-10
US20220090075A1 (en) 2022-03-24
FI3717646T3 (fi) 2025-07-17
MX2020005680A (es) 2020-08-20
CN118291458A (zh) 2024-07-05
US12180480B2 (en) 2024-12-31
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SMT202500334T1 (it) 2025-11-10
EP3717646B1 (en) 2025-04-23
SI3717646T1 (sl) 2025-08-29
EP4328306A3 (en) 2025-05-21
PE20211238A1 (es) 2021-07-09
CN111433361B (zh) 2024-03-29
US20230332152A1 (en) 2023-10-19
EP4328306A2 (en) 2024-02-28
IL274146B2 (en) 2024-11-01
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WO2019109001A1 (en) 2019-06-06
HRP20250804T1 (hr) 2025-09-12
RS66986B1 (sr) 2025-07-31
MX2025009778A (es) 2025-09-02
JP7297320B2 (ja) 2023-06-26
LT3717646T (lt) 2025-06-10
CA3079755A1 (en) 2019-06-06
US11198869B2 (en) 2021-12-14
JP2021505129A (ja) 2021-02-18

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