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Progress Report: 3rd Gen Oligonucleotide Targeting DUX4 in Mouse Model

Posted by George Shaw on January 1, 2018

by Yi-Wen Chen
See grant: Investigation of a third generation oligonucleotide targeting DUX4 using a new mouse model of FSHD

In this study, we are testing an agent (3GA) that can potentially reduce the disease-causing DUX4 in muscles of patients with FSHD. A new mouse model (FLExD mice) that aberrantly expresses DUX4 is used for the study. In our studies, we first showed that the 3GA was able to enter muscles after one subcutaneous injection. We then conducted a study in which the FLExD mice received total six injections, one injection every other day. We showed that the expression of DUX4 was reduced by the 3GA in the muscles of the mice. Our next step is to conduct a longer trial (4 weeks) and to determine whether the muscle function is improved in the treated mice. We will also evaluate safety of the 3GA at the end of the longer trial.