Afrooz Rashnonejad

Background

About Afrooz Rashnonejad

Dr. Rashnonejad’s core expertise is in developing AAV-based gene therapies for rare neurodegenerative and neuromuscular diseases. During her Ph.D, she investigated intra-uterine (aka prenatal or fetal) AAV gene therapy for Spinal Muscular Atrophy (SMA). During her postdoc, she developed CRISPR-Cas13-mediated DUX4-silencing and U7snRNA-mediated exon-skipping approaches for treating Facioscapulohumeral muscular dystrophy (FSHD) as well as characterizing a new FSHD mouse model. In her own translational lab, Dr. Rashnonejad continues developing novel AAV-based gene therapies for rare pediatric disorders with the goal of translating these research programs into the clinic. 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 to achieve tissue- and cell-specific treatments for PNS disorders. Her current research focus has also included evaluating novel gene therapies for Charcot-Marie-Tooth type 1B (CMT1B), mitochondrial DNA depletion syndrome, and Nemaline Myopathy. Rashnonejad lab is also interested in investigating in-utero gene therapies for severe inherited diseases that start prenatally. The team employs cutting-edge technologies such as RNAi and artificial miRNA, and CRISPR approaches, advanced molecular methods, DNA/RNA sequencing, and testing therapies in animal models. Please visit our lab page for more information We are committed to mentoring the next generation of scientists. Rashnonejad lab is always seeking talented and passionate scientists to join the team, from students to postdocs to staff scientists who thrive in a fast-paced challenging research environment. If you have a desire to make a significant impact in rare pediatric neuromuscular and neurological diseases, please contact us and include your CV with a cover letter explaining your research interest and future goal.