Chris and Monique Lorson
Research Interests The Lorson lab is comprised of several research teams largely centered on translational medicine and neurodegenerative diseases including Spinal Muscular Atrophy (SMA), SMA with Respiratory Distress (SMARD), Charcot-Marie-Tooth (CMT), and others. Current projects include: Development of a novel drug for SMA — a splice-switching antisense oligonucleotide — that dramatically extends survival in SMA animal models from a single dose at birth. Identification of genes and pathways that improve the SMA/SMARD phenotypes, such as Plastin-3 or alpha-synuclein and then validating these factors using AAV-mediated gene therapy. Small molecule development in collaboration with the Androphy lab (IUPUI) and the Hodgetts…
