Amyotrophic Lateral Sclerosis (ALS)
Below we provide a few examples of the research being done here at the Center for NeuroGenetics specifically on Amyotrophic Lateral Sclerosis (ALS). Visit each of the individual researchers sites by clicking on their name for more detailed information on their backgrounds and research projects.
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.
Drs. Laura Ranum and Maurice Swanson received received a National Institutes of Health (NIH) grant to understand the molecular pathophysiology and therapeutic efficacy of antisense oligonucleotides for the treatment of C9ORF72 ALS/FTD.
Dr. Laura Ranum received a Muscular Dystrophy Association (MDA) grant for the molecular characterization of a novel C9ORF72 BAC mouse model and the investigation of an antibody therapy strategy in this model.
Dr. Laura Ranum received support from Target Amyotrophic Lateral Sclerosis to develop an immunotherapy strategy using human-derived antibodies that specifically recognize dipeptide repeat proteins (DPRs) of C9ORF72, which accumulate in ALS/FTD patient brains, to determine therapeutic potential.
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.