Nonsense Suppression and Genetic Codon Alteration by Targeted Modification

Xinliang Zhao, Co-Inventor

Patent Number: US 8,603,457

Date issued: December 10, 2013

Methods for affecting mRNA expression or translation through the modification of pre-mRNA or mRNA transcripts are described. In one embodiment of the methods of the present invention, the branch point adenosine of a pre-mRNA transcript is 2'-O-methylated to block splicing and subsequent expression of the protein encoded by the transcript. In another embodiment, a uridine residue in a nonsense stop codon may be modified to pseudouridine, causing the translation machinery to read through the nonsense stop codon and translate a full length protein.

RNA Modification to Modulate Telomerase Activity

Chao Huang, Co-Inventor

Technology ID: 6-1946

2'-O-methylation at U809 reduces telomerase activity resulting in telomere shortening, whereas 2'-O-methylation at A804 or A805 leads to moderate telomere lengthening. Researchers at the University of Rochester have shown that the use of 2'-O-methylation can be accomplished in vivo and have an effect on telomerase activity.

Targeted Pre-mRNA/mRNA Modification and Gene Regulation

Technology ID: 6-1446

This technology describes a novel approach to silencing gene expression by interfering with the processing of its mRNA transcript. This method is advantageous over more traditional methods such as iRNA, because it can be used to silence specific alternatively spliced variants of the gene. This is particularly advantageous relative to the development of human therapeutics as it is believed 50% of human transcripts are alternatively spliced.