MX340397B - Modulacion de expresion de huntingtina. - Google Patents

Modulacion de expresion de huntingtina.

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Publication number
MX340397B
MX340397B MX2012003006A MX2012003006A MX340397B MX 340397 B MX340397 B MX 340397B MX 2012003006 A MX2012003006 A MX 2012003006A MX 2012003006 A MX2012003006 A MX 2012003006A MX 340397 B MX340397 B MX 340397B
Authority
MX
Mexico
Prior art keywords
modulation
huntingtin expression
compositions
compounds
methods
Prior art date
Application number
MX2012003006A
Other languages
English (en)
Other versions
MX2012003006A (es
Inventor
M Freier Susan
Frank Bennett C
Hung Gene
H Cheng Seng
Kordasiewicz Holly
Stanek Lisa
W Cleveland Don
Shihabuddin Lamya
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of MX2012003006A publication Critical patent/MX2012003006A/es
Publication of MX340397B publication Critical patent/MX340397B/es

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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Abstract

La presente invención se refiere a métodos, compuestos y composiciones para reducir la expresión de ARNm y proteína de huntingtina en un animal. Dichos métodos, compuestos, y composiciones son útiles para tratar, prevenir, retrasar, o mejorar la enfermedad de Huntington, o un síntoma de la misma. Un oligonucleótido de cadena individual dirigido a un ácido nucleico que codifica para huntingtina y que puede inhibir la expresión de huntingtina, en el cual el oligonucleótido modificado comprende: un segmento de espacio que consiste de diez desoxinucleósidos ligados; un segmento de ala 5´que consiste de cinco nucleósidos ligados; y un segmento de la 3´que consiste de cinco nucleósidos ligados; en el cual el segmento de espacio está posicionado entre el segmento de ala 5´y el segmento de la 3´, en el cual cada nucleósido de cada segmento de ala comprende un azúcar 2´-O-metoxietilado; en el cual los enlazamientos internucleósido dentro del segmento de espacio, los enlazamientos que conectan el segmento de espacio al segmento de ala 5´o 3´, y los enlazamientos para los nucleósidos más hacia el extremo 5´y más hacia el extremo 3´de cada segmento de ala son todos enlazamientos de fosforotioato, los enlazamientos internucleósido que conectan el resto de los nucleósidos de los segmentos de ala tanto 5´como 3´son enlazamientos de fosfoldiéster; y en el cual todas la scitosinas son 5-metilcitosinas; y en donde la secuencia de nucleobase del iligonucleótido consiste de una secuencia recitada en SEQ ID NO: 22 ó 32.
MX2012003006A 2009-09-11 2010-09-10 Modulacion de expresion de huntingtina. MX340397B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US24185309P 2009-09-11 2009-09-11
PCT/US2010/048532 WO2011032045A1 (en) 2009-09-11 2010-09-10 Modulation of huntingtin expression

Publications (2)

Publication Number Publication Date
MX2012003006A MX2012003006A (es) 2012-06-28
MX340397B true MX340397B (es) 2016-07-07

Family

ID=43732828

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2012003006A MX340397B (es) 2009-09-11 2010-09-10 Modulacion de expresion de huntingtina.
MX2017016415A MX380175B (es) 2009-09-11 2010-09-10 Modulacion de expresion de huntingtina.
MX2016004031A MX353152B (es) 2009-09-11 2012-03-09 Modulación de expresión de huntingtina.

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Application Number Title Priority Date Filing Date
MX2017016415A MX380175B (es) 2009-09-11 2010-09-10 Modulacion de expresion de huntingtina.
MX2016004031A MX353152B (es) 2009-09-11 2012-03-09 Modulación de expresión de huntingtina.

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US (8) US8906873B2 (es)
EP (3) EP3626823A1 (es)
JP (5) JP5809146B2 (es)
KR (5) KR102173836B1 (es)
CN (3) CN102625809B (es)
AU (4) AU2010292003B2 (es)
BR (1) BR112012005448B1 (es)
CA (2) CA2773886C (es)
DK (1) DK2475675T3 (es)
ES (2) ES2772825T3 (es)
HU (1) HUE033113T2 (es)
IL (4) IL218482A (es)
MX (3) MX340397B (es)
NZ (3) NZ599032A (es)
PL (1) PL2475675T3 (es)
PT (1) PT2475675T (es)
RU (2) RU2751847C9 (es)
SI (1) SI2475675T1 (es)
WO (1) WO2011032045A1 (es)

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