Rashnonejad Lab

The Rashnonejad Lab focuses on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative and neuromuscular diseases. Its team works on monogenetic disorders including Charcot-Marie-Tooth type 1B (CMT1B), Dejerine–Sottas syndrome (DSS, another form of CMT disorder), Smith-Kingsmore syndrome (SKS) and several mitochondrial disorders. The Rashnonejad Lab is also interested in developing in utero (prenatal or fetal) gene therapies for severe, congenital neuromuscular disorders. The lab uses cutting-edge technologies such as gene replacement, gene silencing (RNAi), genome editing (CRSPR) and Exon skipping and facilitates their translation into human testing by optimizing AAV vector delivery to target tissues. The team’s research requires the use of cellular and rodent models to investigate disease pathogenesis, vector safety and efficacy, and the identification of biomarkers as therapeutic outcome measures toward translating these therapies to the clinic.

The team’s main research interests are:

  • Developing gene therapies for inherited pediatric disorders
  • Developing gene therapies for peripheral neuropathies
  • Engineering AAV vectors and selective targeting of these vectors to the central and peripheral nervous systems
  • Developing in-utero (aka prenatal or fetal) gene therapies

Lab Staff

Afrooz Rashnonejad

Afrooz Rashnonejad, MSc, PhD
Principal Investigator

Afrooz Rashnonejad, MSc, PhD, is an assistant professor in the Department of Pediatrics at the Ohio State University College of Medicine and principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital. Dr. Rashnonejad is the chair of the American Society of Gene and Cell therapy (ASGCT)’s New Investigator Committee and she was twice awarded the “Excellence in Research Award” at the ASGCT annual meetings. She was also honored with the President’s Prize at the 24th annual meeting of the World Muscle Society in 2019.

Dr. Rashnonejad earned her PhD in Biotechnology from Ege University, Turkey, in 2017, after completing two Master’s of Science degrees in Microbiology, and Stem Cell. During her PhD, she developed an in-utero (AKA prenatal or fetal) gene therapy for Spinal Muscular Atrophy (SMA). During this period, she also worked in Dr. Guangping Gao (her PhD co-advisor)’s lab in the Center for Gene Therapy at the University of Massachusetts Medical School as a visiting scientist to master large-scale recombinant AAV production. She then completed postdoctoral training in the lab of Scott Harper, PhD, at the Center for Gene Therapy at Nationwide Children’s Hospital. During her postdoc, Dr. Rashnonejad developed RNA targeting AAV.CRISPR-Cas13 and U7-snRNA mediated exon skipping to treat autosomal dominant inherited Facioscapulohumeral Muscular Dystrophy (FSHD).

Nationwide Children's Research Staff

Jingting Zhu, PhD
Research Associate and Lab Manager

Jingting Zhu, PhD, joined Rashnonejad Lab in 2022. Her work focuses on developing Schwann cell specific AAV vectors toward developing gene therapies for peripheral neuropathies such as CMT1B, which is caused by mutations of the myelin protein zero (MPZ) gene.

Dr. Zhu received her bachelor’s and doctorate degrees in Biological Sciences at Liverpool University. Her postdoctoral research at the Center for Vaccine and Immunity at Nationwide Children’s Hospital focused on the pathogenesis associated with hepatitis virus. Outside of the lab, she enjoys watching movies and hiking.

Nationwide Children's Research Staff

Esra Goktas, MD
Research Associate

Esra Goktas, MD, joined the Rashnonejad Lab in 2022. Her current research focuses on developing various ex vivo models of peripheral neuropathies to investigate the efficacy of gene therapies before testing them in animal models. She also develops intrauterine prenatal gene therapies for severe congenital neurological disorders.

Dr. Goktas earned her medical degree in 2016 from the University of Health Sciences, Gulhane Faculty of Medicine, Turkey. In her previous work, she used CRISPR-Cas9 genome editing to prevent immune responses to transplanted tissues in large animal models. Outside of the lab, her hobbies include biking and playing chess.

Nationwide Children's Research Staff

Russell Chuah
Research Assistant

Russell’s current research focuses on developing novel gene therapies for mitochondrial disorders affecting brain and muscles. He received his bachelor’s degree in Molecular Genetics from The Ohio State University in 2022, and he hopes to begin graduate school after acquiring lab experience and beginning to publish papers.

Nationwide Children's Research Staff

Kimberly Jordan
Administrative Support III

Kim, a certified executive assistant professional with a background of 22 years of administrative experience, is responsible for performing a wide variety of complex administrative tasks for faculty in the Center for Gene Therapy. She joined the team in 2022.

Outside of work, she enjoys spending time with her daughters and friends, and reading.

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