DOP2017000185A - ARNI VARIANTE - Google Patents
ARNI VARIANTEInfo
- Publication number
- DOP2017000185A DOP2017000185A DO2017000185A DO2017000185A DOP2017000185A DO P2017000185 A DOP2017000185 A DO P2017000185A DO 2017000185 A DO2017000185 A DO 2017000185A DO 2017000185 A DO2017000185 A DO 2017000185A DO P2017000185 A DOP2017000185 A DO P2017000185A
- Authority
- DO
- Dominican Republic
- Prior art keywords
- rnai
- recombinant
- guide sequence
- huntington
- disease
- Prior art date
Links
- 241000320892 Clerodendrum phlomidis Species 0.000 title 1
- 229940125365 angiotensin receptor blocker-neprilysin inhibitor Drugs 0.000 title 1
- 108091030071 RNAI Proteins 0.000 abstract 4
- 230000009368 gene silencing by RNA Effects 0.000 abstract 4
- 208000023105 Huntington disease Diseases 0.000 abstract 2
- 238000003776 cleavage reaction Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- 230000007017 scission Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Se proporcionan en la presente memoria moléculas de ARNi que incluyen una primera cadena que contiene una secuencia guía y una segunda cadena que comprende una secuencia no guía en donde la secuencia no guía contiene una protuberancia opuesta a la región semilla de las secuencias guía; p. ej., opuesta a la secuencia de escisión. En algunos aspectos, la invención proporciona ARNi para el tratamiento de la enfermedad de Huntington. Adicionalmente se proporcionan en la presente memoria casetes de expresión, vectores (p. ej., vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y de HSV recombinantes), células, partículas virales, y composiciones farmacéuticas que contienen el ARNi. También se proporcionan adicionalmente en la presente memoria métodos y kits relacionados con el uso del ARNi, por ejemplo, para el tratamiento de la enfermedad de Huntington. CIP: C12N 15/113RNAi molecules are provided herein that include a first chain containing a guide sequence and a second chain comprising a non-guide sequence wherein the non-guide sequence contains a protrusion opposite the seed region of the guide sequences; p. eg, opposite to the cleavage sequence. In some aspects, the invention provides RNAi for the treatment of Huntington's disease. Additionally, expression cassettes, vectors (eg, rAAV vectors, recombinant adenovirals, recombinant lentivirals, and recombinant HSVs), cells, viral particles, and pharmaceutical compositions containing the RNAi are provided herein. Methods and kits related to the use of RNAi are also provided herein, for example, for the treatment of Huntington's disease. CIP: C12N 15/113
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201561114578P | 2015-02-10 | 2015-02-10 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DOP2017000185A true DOP2017000185A (en) | 2017-09-29 |
Family
ID=59973997
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DO2017000185A DOP2017000185A (en) | 2015-02-10 | 2017-08-09 | ARNI VARIANTE |
Country Status (1)
| Country | Link |
|---|---|
| DO (1) | DOP2017000185A (en) |
-
2017
- 2017-08-09 DO DO2017000185A patent/DOP2017000185A/en unknown
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