Modulation of pre-mrna using splice modulating...

Details Modulation of pre-mrna using splice modulating... Modulation of pre-mrna using splice modulating...

C - Chemistry, Metallurgy

12

N

C12N 15/113 (2010.01) A61K 31/7105 (2006.01) C07H 21/02 (2006.01)

Patent

CA 2750379

The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.

L'invention concerne une catégorie de composés appelés oligonucléotides de modulation d'épissage (SMO) modulant l'épissage de pré-ARNm, ce qui modifie l'expression et la fonctionnalité d'une protéine spécifique dans une cellule. L'invention concerne également des compositions et des méthodes de modulation d'épissage de pré-ARNm au moyen d'un SMO selon l'invention, pour supprimer les mutations pathogènes dans une protéine. Ainsi, la présente invention concerne des compositions et des méthodes permettant de traiter un patient à risque ou susceptible de développer une maladie, affection ou trouble associé à l'expression ou l'activité aberrante ou indésirable de pré-ARNm cibles.

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