The Saad Lab conducts cutting-edge research to understand the causes of genetic disorders, including neuromuscular diseases, and develops innovative techniques to diagnose, treat and cure these conditions. Specifically, Nizar Saad, MSc, PhD, and his team are dedicated to developing new gene therapy approaches and bringing them to the clinic and to identifying new blood biomarkers for diagnosis, prediction of disease progression and evaluation of therapy effectiveness.
Our Areas of Focus
- The identification of novel non-invasive biomarkers by investigating the molecular content of extracellular vesicles found in biofluids such as blood.
- The development of viral (adeno-associated virus) and non-viral (extracellular vesicle) based gene therapies for genetic diseases.
The developed approaches are currently focused on microRNA- and CRISPR-Cas-based knockdown therapies. The Saad lab is also developing novel approaches and platforms to enhance tissue specificity, efficacy and potency of viral and non-viral gene therapies.
While the Saad Lab focuses on diseases including facioscapulohumeral muscular dystrophy (FSHD), Duchenne muscular dystrophy (DMD), Emery-Dreifuss muscular dystrophy (EDMD), dilated cardiomyopathies (DCM) with LMNA mutations and progeria, the developed technologies could be applied to other diseases. Dr. Saad and his team hope their discoveries can help a wide range of patients with genetic conditions.
Collaborate With Us
The Saad Lab engages with a variety of collaborators across Nationwide Children’s, as well as labs from the The Ohio State University, the University of Rochester and TGen City of Hope in Phoenix.
The Saad Lab is always open to new collaborations.
Learn More About Working in the Center for Gene Therapy
Throughout his career, Dr. Saad has trained many research technicians, graduate students and postdoctoral fellows, and the Saad Lab is always seeking to hire passionate and motivated scientists.
- Saad’s trainees benefit from his experience in RNA biology, with opportunities to enhance their molecular biology and biochemistry skills and study, design and use RNA sensing molecules in in vitro applications.
- They also benefit from his experience in designing and using miRNA-based gene therapy knockdown tools in in vitro and in vivo settings to inhibit the expression of gain-of-function mutations.
- Team members explore new areas of research involving the use of exosomes for biomarker discovery and gene therapy.
The Saad Lab embraces a culture of collaboration and inclusivity to drive innovative research and foster a supportive community where everyone's unique perspectives and contributions are valued and respected.
Connect With Our Team
The Saad Lab is hiring passionate and motivated scientists and open to new collaborations.
Donate to the Saad Lab
Donations to the Saad Lab support research at Nationwide Children's Hospital to accelerate the translation of gene therapies to the clinic. Donations provide funding for hiring talented team members and acquiring the supplies, reagents and equipment necessary for research projects.
Meet Our Team
Nizar Y. Saad, MSc, PhD
Nizar Saad, MSc, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor in the Department of Pediatrics at The Ohio State University College of Medicine. Dr. Saad's expertise is in RNA biology, gene therapy and their respective translational applications. His research interests are understanding the pathobiology of neuromuscular disorders, the discovery of circulating exosome-based biomarkers and the development of viral and non-viral based gene therapies towards these disorders and other rare genetic diseases (i.e., progeria). Dr. Saad’s growing international reputation is demonstrated by his service on the New Investigator, Musculo-Skeletal Gene & Cell Therapy and Oligonucleotide and RNAi Therapeutics committees of the American Society of Gene and Cell Therapy.
Bilal joined the Saad Lab in June 2023. His work focuses on identifying EV-associated molecular signatures of neuromuscular diseases to help track disease onset and progression and applying this knowledge to potential gene therapies. His research interests also include single-molecule imaging. Prior to joining the Saad Lab, he worked as a postdoctoral scholar at the Ohio State University working on the role of RNA modifications in cardiomyocytes. Previously, he obtained his PhD degree at University of Lausanne in Switzerland where he focused on the (sub)cellular transfer of small non-coding RNAs in rodent models of diabetes.
Neel D. Lachhwani
Neel Lachhwani joined the lab in 2022 after earning his bachelor’s degree in Neuroscience from The Ohio State University, where he was inspired to pursue research by studying medical treatment approaches. Neel is interested in supporting the development of life-changing gene therapy approaches for children and adults with neuromuscular diseases. In his free time, Neel volunteers as a reading, science and math tutor for elementary students in underserved communities. Neel also has a passion for sports and enjoys rock climbing and playing soccer.
Administrative Assistant III
Rachael joined Nationwide Children’s in 2022 after working for 12 years in the retail pharmacy industry. In her free time, she likes to take hikes with her dog, Cooper, attend concerts, and tackle different projects and crafts.