Saad Lab
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.
Inside Saad Lab
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.
Connect With Our Team
The Saad Lab engages with a variety of collaborators across Nationwide Children’s, as well as labs from 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 Jerry R. Mendell 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.
