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MX2015016588A - Estructura, fabricacion y usos de los peptidos permeables a las celulas, derivados de humanos, conjugados con los peptidos de carga especificos, biologicamente activos. - Google Patents

Estructura, fabricacion y usos de los peptidos permeables a las celulas, derivados de humanos, conjugados con los peptidos de carga especificos, biologicamente activos.

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Publication number
MX2015016588A
MX2015016588A MX2015016588A MX2015016588A MX2015016588A MX 2015016588 A MX2015016588 A MX 2015016588A MX 2015016588 A MX2015016588 A MX 2015016588A MX 2015016588 A MX2015016588 A MX 2015016588A MX 2015016588 A MX2015016588 A MX 2015016588A
Authority
MX
Mexico
Prior art keywords
peptides
human
conjugates
fusion proteins
biologically active
Prior art date
Application number
MX2015016588A
Other languages
English (en)
Inventor
Bruce H Littman
John F Thompson
Frank W Marcoux
Original Assignee
Portage Pharmaceuticals Ltd
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 Portage Pharmaceuticals Ltd filed Critical Portage Pharmaceuticals Ltd
Publication of MX2015016588A publication Critical patent/MX2015016588A/es

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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013Iduronate-2-sulfatase (3.1.6.13)

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Abstract

La presente invención se refiere a composiciones para los conjugados, incluyendo proteínas de fusión, y métodos de usarlos para tratar una variedad de condiciones. En algunas modalidades, los conjugados y/o proteínas de fusión incorporan un homeodominio humano de 60-amino ácidos (por ejemplo, péptidos derivados de genes HOX humanos), para translocar los péptidos y proteínas funcionales y regulatarias u otras moléculas biológicamente activas tales como ácidos nucleicos, que no están asociados naturalmente con el homeodominio humano, a través de la célula y las membranas nucleares destinados a sitios de acción sin provocar una respuesta inmune no deseada que puede reducir la exposición al conjugado y/o dar lugar a un evento adverso clínico. En realizaciones adicionales, se da a conocer conjugados y proteínas de fusión pueden pasar a través de la barrera sangre-cerebro para permitir la entrada en el SNC. En diversas realizaciones, las composiciones descritas son adecuadas para la entrega en una célula (i) el producto de expresión de un gen de interés y/o (ii) nuevos péptidos o polinucleótidos para regular la función génica.
MX2015016588A 2013-06-11 2014-06-11 Estructura, fabricacion y usos de los peptidos permeables a las celulas, derivados de humanos, conjugados con los peptidos de carga especificos, biologicamente activos. MX2015016588A (es)

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