Modulation of pre-MRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
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United States of America Patent
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Feb 6, 2018
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May 20, 2016
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Jan 14, 2009
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Abstract
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.
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