AR123066A1 - COMPOUNDS AND METHODS TO REDUCE THE EXPRESSION OF APP - Google Patents
COMPOUNDS AND METHODS TO REDUCE THE EXPRESSION OF APPInfo
- Publication number
- AR123066A1 AR123066A1 ARP210102093A ARP210102093A AR123066A1 AR 123066 A1 AR123066 A1 AR 123066A1 AR P210102093 A ARP210102093 A AR P210102093A AR P210102093 A ARP210102093 A AR P210102093A AR 123066 A1 AR123066 A1 AR 123066A1
- Authority
- AR
- Argentina
- Prior art keywords
- app
- compounds
- methods
- modified oligonucleotide
- modified
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 206010002942 Apathy Diseases 0.000 abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- 208000037259 Amyloid Plaque Diseases 0.000 abstract 1
- 208000028698 Cognitive impairment Diseases 0.000 abstract 1
- 206010010904 Convulsion Diseases 0.000 abstract 1
- 206010012289 Dementia Diseases 0.000 abstract 1
- 206010017577 Gait disturbance Diseases 0.000 abstract 1
- 230000002159 abnormal effect Effects 0.000 abstract 1
- 208000013404 behavioral symptom Diseases 0.000 abstract 1
- 208000010877 cognitive disease Diseases 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001771 impaired effect Effects 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000002777 nucleoside Substances 0.000 abstract 1
- 125000003835 nucleoside group Chemical group 0.000 abstract 1
- 230000000750 progressive effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o la actividad del ARN de APP en una célula o animal y, en determinadas instancias, para reducir la cantidad de proteína APP en una célula o animal. Dichos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o una característica de una enfermedad o trastorno neurodegenerativo. Dichos síntomas y características incluyen deterioro cognitivo, lo que incluye un deterioro de la memoria y las aptitudes lingüísticas, síntomas psicológicos y del comportamiento tales como apatía y falta de motivación, perturbación de la marcha y crisis epilépticas, demencia progresiva y depósitos amiloides anómalos. Un compuesto oligomérico que comprende un oligonucleótido modificado que consiste en 12 a 30 nucleósidos enlazados en donde la secuencia de nucleobases del oligonucleótido modificado es al menos 80% complementaria a una parte de igual longitud de un ácido nucleico de APP y en donde el oligonucleótido modificado comprende al menos una modificación seleccionada de un resto de azúcar modificado y un enlace internucleósido modificado.Compounds, methods, and pharmaceutical compositions are provided for reducing the amount or activity of APP RNA in a cell or animal and, in certain instances, for reducing the amount of APP protein in a cell or animal. Said compounds, methods and pharmaceutical compositions are useful for ameliorating at least one symptom or characteristic of a neurodegenerative disease or disorder. Such symptoms and features include cognitive impairment, including impaired memory and language skills, psychological and behavioral symptoms such as apathy and lack of motivation, gait disturbance and seizures, progressive dementia, and abnormal amyloid deposits. An oligomeric compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides wherein the nucleobase sequence of the modified oligonucleotide is at least 80% complementary to an equal length portion of an APP nucleic acid and wherein the modified oligonucleotide comprises at least one modification selected from a modified sugar moiety and a modified internucleoside linkage.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063057816P | 2020-07-28 | 2020-07-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR123066A1 true AR123066A1 (en) | 2022-10-26 |
Family
ID=83835139
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210102093A AR123066A1 (en) | 2020-07-28 | 2021-07-28 | COMPOUNDS AND METHODS TO REDUCE THE EXPRESSION OF APP |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR123066A1 (en) |
-
2021
- 2021-07-28 AR ARP210102093A patent/AR123066A1/en unknown
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|---|---|---|---|
| FB | Suspension of granting procedure |