Ptc Therapeutics, Inc.

Jin Zhuo

Ellen Welch

Nikolai A Naryshkin

Charles Romfo

Meenal Patel

Westley J Friesen

Yuki Tomizawa

The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular mystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).

Methods of screening for compounds for treating muscular dystrophy using human ALPHA7 integrin mRNA translation regulation

The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular dystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).

Methods of screening for compounds for treating muscular dystrophy using UTRN mRNA translation regulation

The present invention relates to methods for identifying compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay by screening and identifying compounds that modulate the post-transcriptional expression of any gene with a premature translation stop codon. The invention particularly relates to using any gene encoding a premature stop codon to identify compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay. A compound that modulates premature translation termination and/or nonsense-mediated mRNA decay of a target gene is identified using standard methods known in the art to measure changes in translation or mRNA stability of the gene product or mRNA of the gene with the premature stop codon. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.

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Belle Mead, NJ, United States of America

Jin Zhuo