Ptc Therapeutics, Inc.

Charles Romfo

Nikolai A Naryshkin

Ellen Welch

Liangxian Cao

Christopher Robert Trotta

Meenal Patel

Westley J Friesen

Yuki Tomizawa

Thomas W Davis

Panayiota Trifillis

Jin Zhuo

Matthew C Pellegrini

Marla L Weetall

Langdon Miller

Samit Hirawat

Sergey Paushkin

Harry H Miao

Anuradha Mehta

Jin Zhou

Anuradha Roy

The invention relates to the fields of screening assays, compounds, and methods for altering gene expression and protein levels. In particular, the invention includes assays to screen for agents capable of modulating gene expression in a UTR-dependent manner and agents capable of modulating gene expression.

Methods and agents for screening for compounds capable of modulating gene expression

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

Methods of screening for compounds for treating muscular dystrophy using mIGF1 mRNA translation regulation

Methods for treating prostate cancer involving the administration of a compound that selectively inhibits pathological production of human vascular endothelial growth factor (VEGF) are described. Methods for treating benign prostatic hyperplasia involving the administration of a compound that selectively inhibits pathological production of human VEGF are also described. The compound may be administered as a single-agent therapy or in combination with one or more additional therapies to a human in need of such treatment.

Methods for treating prostate conditions

The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.

Methods for treating spinal muscular atrophy

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

In accordance with the present invention, methods for inhibiting the translation of VEGF and methods for decreasing VEGF level by inhibiting VEGF translation are provided. In another aspect of the invention, compounds that inhibit the 5'-UTR-dependent translation of VEGF and methods for identifying such compounds are provided.

Inhibition of VEGF translation

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Easton, PA, United States of America

Charles Romfo