AR118517A1 - COMPOUNDS AND METHODS FOR MODULAR UBE3A-ATS - Google Patents
COMPOUNDS AND METHODS FOR MODULAR UBE3A-ATSInfo
- Publication number
- AR118517A1 AR118517A1 ARP200100866A ARP200100866A AR118517A1 AR 118517 A1 AR118517 A1 AR 118517A1 AR P200100866 A ARP200100866 A AR P200100866A AR P200100866 A ARP200100866 A AR P200100866A AR 118517 A1 AR118517 A1 AR 118517A1
- Authority
- AR
- Argentina
- Prior art keywords
- ube3a
- compounds
- methods
- ats
- subject
- Prior art date
Links
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o la actividad de UBE3A-ATS, la transcripción anti sentido endógena de la proteína de ubiquitina ligasa E3A (UBE3A) en una célula o sujeto, y en determinados casos aumentar la expresión de la UBE3A progenitora y la cantidad de proteína UBE3A en una célula o sujeto. Tales compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica de un trastorno neurogenético. Dichos síntomas y características incluyen retrasos en el desarrollo, ataxia, impedimento de habla, problemas del sueño, convulsiones, y anormalidades de EEG. Tales trastornos neurogenéticos incluyen el síndrome de Angelman.Compounds, methods and pharmaceutical compositions are provided to reduce the amount or activity of UBE3A-ATS, the endogenous antisense transcription of the ubiquitin ligase protein E3A (UBE3A) in a cell or subject, and in certain cases increase the expression of the Parent UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful for ameliorating at least one symptom or feature of a neurogenetic disorder. Such symptoms and features include developmental delays, ataxia, speech impediment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman syndrome.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962826521P | 2019-03-29 | 2019-03-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR118517A1 true AR118517A1 (en) | 2021-10-20 |
Family
ID=78803824
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200100866A AR118517A1 (en) | 2019-03-29 | 2020-03-27 | COMPOUNDS AND METHODS FOR MODULAR UBE3A-ATS |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR118517A1 (en) |
-
2020
- 2020-03-27 AR ARP200100866A patent/AR118517A1/en unknown
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