Keyword Analysis & Research: readthrough compounds

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What is the read-through inducing compound tc007 used for?

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum Mol Genet. 2009;18:3906–13. [PMC free article][PubMed] [Google Scholar] 84. McCaughan KK, Brown CM, Dalphin ME, Berry MJ, Tate WP.

What do we know about the readthrough properties of sri-37240?

SRI-37240 was one of the compounds in the Enamine library (T6740530). Notably, we were not aware of the previous patent from Novartis regarding the readthrough properties of this compound at the time of the screen.

Can compound-induced readthrough of premature termination codons help treat nonsense mutations?

Large numbers of genetic disorders are caused by nonsense mutations for which compound-induced readthrough of premature termination codons (PTCs) might be exploited as a potential treatment strategy.

What is the role of stopstop codon read-through with PTC124?

Stop codon read-through with PTC124 induces palmitoylprotein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients. Mol Genet Metab. 2011;104:338–45.

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