The following CNG faculty have been successful in securing new funding for various research projects. Below is a brief summary of the new grants awarded in 2017.
Drs. Andy Berglund, Maurice Swanson and Eric Wang received a National Institutes of Health (NIH) grant to determine the cis and trans acting mechanisms through which the splicing factor, muscleblind, regulates alternative splicing. Results from these studies will provide a better understanding of muscleblind’s role in myotonic dystrophy.
Dr. Andy Berglund received a Muscular Dystrophy Association (MDA) grant to determine the mechanism of action (MOA) of a class of lead compounds that rescue missplicing in myotonic dystrophy. Understanding the MOA will be used to direct the synthesis of new molecules for study in myotonic dystrophy cell models which could lead to new therapeutic treatments for myotonic dystrophy.
Dr. Maurice Swanson received a Muscular Dystrophy Association (MDA) grant to develop knockout and transgenic models to extend the RNA dominance hypothesis to congenital myotonic dystrophy.
Dr. Laura Ranum received support from Target Amyotrophic Lateral Sclerosis (ALS) to develop an immunotherapy strategy using human-derived antibodies that specifically recognize dipeptide repeat proteins (DPRs) of C9ORF72, which accumulate in ALS/FTD, to determine therapeutic efficacy.
Dr. Laura Ranum received support from Target Amyotrophic Lateral Sclerosis (ALS) to investigate nuclear transport related defects as a pathophysiological event in C9ORF72 and sporadic ALS.
Drs. Laura Ranum and Eric Wang received support from Target Amyotrophic Lateral Sclerosis (ALS) to investigate the therapeutic potential of CRISPR-Cas9 in C9ORF72 ALS/FTD.