Dr. Rashnonejad’s core expertise is in developing AAV-based gene therapies for rare neurodegenerative and neuromuscular diseases. Although AAV vectors appear to be the safest and most effective delivery vehicles; however, they do not come with an innate tropism toward a specific tissue/cell type. Targeting the peripheral nerve system (PNS) is still challenging using AAV vectors and is the most significant barrier to developing gene therapy for peripheral neuropathies. Her team is working to address this need by designing next-generation AAV vectors using capsid engineering to achieve tissue- and cell-specific treatments for PNS disorders. Her current research focus has also included evaluating novel AAV-based gene therapies for Charcot-Marie-Tooth type 1B (CMT1B), Neurodegenerative-mitochondrial DNA depletion syndromes, and Nemaline Myopathy. The Rashnonejad lab is also interested in investigating in-utero (aka prenatal or fetal) gene therapies for severe inherited diseases that start prenatally. The team employs cutting-edge technologies such as artificial miRNA and CRISPR approaches, advanced molecular methods, DNA/RNA sequencing, and testing therapies in animal models.