EA202192403A1 - CONNECTIONS AND METHODS OF MODULATION UBE3A-ATS - Google Patents
CONNECTIONS AND METHODS OF MODULATION UBE3A-ATSInfo
- Publication number
- EA202192403A1 EA202192403A1 EA202192403A EA202192403A EA202192403A1 EA 202192403 A1 EA202192403 A1 EA 202192403A1 EA 202192403 A EA202192403 A EA 202192403A EA 202192403 A EA202192403 A EA 202192403A EA 202192403 A1 EA202192403 A1 EA 202192403A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- ube3a
- methods
- ats
- compounds
- subject
- Prior art date
Links
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Предложены соединения, способы и фармацевтические композиции для снижения количества или активности UBE3A-ATS, эндогенного антисмыслового транскрипта убиквитинпротеинлигазы E3A (UBE3A) в клетке или в организме субъекта, и в определенных случаях повышения экспрессии исходной UBE3A и количества белка UBE3A в клетке или в организме субъекта. Такие соединения, способы и фармацевтические композиции применимы для ослабления по меньшей мере одного симптома или отличительного признака нейрогенетического расстройства. Такие симптомы и отличительные признаки включают задержку развития, атаксию, нарушение речи, проблемы со сном, судороги и нарушения на ЭЭГ. Такие нейрогенетические расстройства включают синдром Ангельмана.Compounds, methods, and pharmaceutical compositions are provided to reduce the amount or activity of UBE3A-ATS, an endogenous antisense E3A ubiquitin protein ligase (UBE3A) transcript in a cell or body of a subject, and in certain cases, increase the expression of the original UBE3A and the amount of UBE3A protein in a cell or body of a subject. Such compounds, methods, and pharmaceutical compositions are useful in alleviating at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delay, ataxia, speech disturbance, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman syndrome.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962877765P | 2019-07-23 | 2019-07-23 | |
| PCT/US2020/025110 WO2020205463A1 (en) | 2019-03-29 | 2020-03-27 | Compounds and methods for modulating ube3a-ats |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EA202192403A1 true EA202192403A1 (en) | 2021-12-30 |
Family
ID=80631178
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA202192403A EA202192403A1 (en) | 2019-07-23 | 2020-03-27 | CONNECTIONS AND METHODS OF MODULATION UBE3A-ATS |
Country Status (1)
| Country | Link |
|---|---|
| EA (1) | EA202192403A1 (en) |
-
2020
- 2020-03-27 EA EA202192403A patent/EA202192403A1/en unknown
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