MX390355B - Regulacion de la expresión génica a través de la modulación mediada por un aptámero del empalme alternativo. - Google Patents

Regulacion de la expresión génica a través de la modulación mediada por un aptámero del empalme alternativo.

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Publication number
MX390355B
MX390355B MX2017009953A MX2017009953A MX390355B MX 390355 B MX390355 B MX 390355B MX 2017009953 A MX2017009953 A MX 2017009953A MX 2017009953 A MX2017009953 A MX 2017009953A MX 390355 B MX390355 B MX 390355B
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Mexico
Prior art keywords
target gene
aptamer
regulation
ligand
gene expression
Prior art date
Application number
MX2017009953A
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English (en)
Other versions
MX2017009953A (es
Inventor
Alex R Boyne
F Oliver Danos
J Michael Volles
Xuecui Guo
Original Assignee
Meiragtx Uk Ii Ltd
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Publication date
Application filed by Meiragtx Uk Ii Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of MX2017009953A publication Critical patent/MX2017009953A/es
Publication of MX390355B publication Critical patent/MX390355B/es

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    • A61K38/44—Oxidoreductases (1)
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
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    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
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Abstract

La presente invención se refiere a una plataforma y métodos para usar la plataforma para la regulación de la expresión de un gen diana usando la exposición a un aptámero-ligando (por ejemplo, una molécula pequeña) La plataforma está provista de un cassette de regulación de genes de polinucleótido que está colocado en el gen diana e incluye un riborregulador sintético posicionado en el contexto de un intrón 5´-exon alternativo-intrón 3´. El riboregulador consta de una región efectora y una región sensora (por ejemplo, un aptámero que se une a una molécula pequeña, un ligando) de modo que el exón alternativo sea empalmado en el ARN mensajero del gen diana cuando el ligando no está presente previniendo así la expresión del gen diana. Cuando el ligando está presente, el exón alternativo no es empalmado en el ARN mensajero del gen diana proporcionando así la expresión del gen diana.
MX2017009953A 2015-02-02 2016-02-02 Regulacion de la expresión génica a través de la modulación mediada por un aptámero del empalme alternativo. MX390355B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562110919P 2015-02-02 2015-02-02
PCT/US2016/016234 WO2016126747A1 (en) 2015-02-02 2016-02-02 Regulation of gene expression by aptamer-mediated modulation of alternative splicing

Publications (2)

Publication Number Publication Date
MX2017009953A MX2017009953A (es) 2018-04-26
MX390355B true MX390355B (es) 2025-03-20

Family

ID=56564618

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017009953A MX390355B (es) 2015-02-02 2016-02-02 Regulacion de la expresión génica a través de la modulación mediada por un aptámero del empalme alternativo.
MX2022002634A MX2022002634A (es) 2015-02-02 2017-08-01 Regulacion de la expresion genica a traves de la modulacion mediada por un aptamero del empalme alternativo.

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MX2022002634A MX2022002634A (es) 2015-02-02 2017-08-01 Regulacion de la expresion genica a traves de la modulacion mediada por un aptamero del empalme alternativo.

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US (4) US10494646B2 (es)
EP (2) EP3265563B1 (es)
JP (3) JP6871174B2 (es)
KR (2) KR102892076B1 (es)
CN (2) CN114990143B (es)
AU (3) AU2016215454B2 (es)
BR (1) BR112017016639A2 (es)
CY (1) CY1124189T1 (es)
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EA (2) EA202092665A3 (es)
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HR (1) HRP20210854T1 (es)
HU (1) HUE054624T2 (es)
IL (2) IL253786B (es)
LT (1) LT3265563T (es)
MX (2) MX390355B (es)
MY (1) MY186203A (es)
NZ (1) NZ735054A (es)
PH (2) PH12021552268A1 (es)
PL (1) PL3265563T3 (es)
PT (1) PT3265563T (es)
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SG (1) SG11201706297RA (es)
SI (1) SI3265563T1 (es)
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