Research Projects

Graphic portrayal of Dr. Martin's research project

Project 1

Led by Paul Marin, PhD, Research Project 1 of our MDSRC Program is dedicated to completing remaining pre-clinical proof of concept and safety studies required to file a pre-IND application with the FDA for a first-in-class bicistronic AAV gene therapy for patients with Limb Girdle Muscular Dystrophy Type R9 (LGMDR9).

Virally-delivered U7snRNAs that target exons can induce significant exon skipping. In this example, the U7snRNA targets exon 2 within the mouse Dup2 model carrying a duplication of that exon. Complete skipping of exon 2 can still result in dystrophin expression via use of a downstream translational initiation site.

Project 2

Led by Kevin Flanigan, MD, Research Project 2 of our MDSRC Program has a long-term goal to develop vectors that maximize RNA splice alteration efficiency to provide the best potential outcome for individual patients.

Project 3

Led by Nizar Saad, PhD, Research Project 3 of our MDSRC Program is aimed to identify DMD circulating EV biomarkers and develop EV-enveloped AAV gene therapy for DMD to shield AAV from anti-AAV antibodies and to increase its transduction efficiency and therapeutic potency.