Featured Researcher – Nizar Y. Saad, PhD
Featured Researcher – Nizar Y. Saad, PhD https://pediatricsnationwide.org/wp-content/themes/corpus/images/empty/thumbnail.jpg 150 150 Gina Vitale https://secure.gravatar.com/avatar/?s=96&d=mm&r=g
Not all genetic diseases are inherited; however, they are all the result of DNA mutations or flaws. Genetic research and gene therapy are integral in finding cures or helpful treatments for diseases such as muscular dystrophy. At the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital, research scientists are engaged in precedent-setting transitional gene therapy for the betterment of children with rare genetic and neuromuscular degenerative diseases.
Nizar Y. Saad, PhD, is an RNA biologist and gene therapy specialist displaying a refreshing enthusiasm for his work. He is a research assistant professor at The Ohio State University College of Medicine and a principal investigator at the Jerry R. Mendell Center for Gene Therapy at the AWRI.
Dr. Saad’s scholastic and professional journey has been impressive. His scientific study of RNA has led to focusing on developing innovative gene therapy approaches for the treatment of diseases such as Facioscapulohumeral muscular dystrophy (FSHD), allowing him to bridge the gap between fundamental science and clinical application. Dr. Saad is committed to advancing therapies that make a meaningful impact on patients’ lives.
Read on to learn more about Dr. Saad’s work and research career.
How/why did you decide to pursue a career in your field?
My journey into science was deeply inspired by the remarkable legacy of my distant relative, Dr. Edmond J. Yunis, MD, a world-renowned scientist and Professor of Pathology at Harvard Medical School. Dr. Yunis was born in Colombia to a Lebanese family who had immigrated there. He and four brothers earned medical degrees and built successful careers in medicine, teaching and research. Their achievements ignited my pursuit of a scientific path.
My father was equally pivotal in shaping my career. He spoke about the extraordinary accomplishments of the Yunis brothers, leaving a lasting impression and encouraging me to dream big and make a difference.
My innate curiosity to understand the world, a deep desire to leave my mark, and familial inspirations clarified my path toward a career in science.
What was your path to your current role?
My scientific journey began in Lebanon, where I earned my undergraduate degree in biochemistry from the Lebanese University in Beirut. I moved to Strasbourg, France, to study at the University of Strasbourg, where I obtained both my master’s and PhD degrees in biochemistry, molecular and structural biology.
During my PhD, I studied riboswitch RNAs and their role in regulating bacterial gene expression. I then joined, as a postdoc, Dr. Tina Henkin’s lab at OSU. Dr. Henkin, amember of the National Academy of Sciences, independently discovered riboswitch RNAs. In her lab, I investigated tRNA-sensing (T box) riboswitch RNA, which are promising targets for the development of a new generation of antibiotics.
Eager to broaden my horizons, I shifted toward translational research, where I could apply my novel ideas to develop therapeutic strategies that make a meaningful impact on patients’ lives. This desire led to my second postdoctoral position in the lab of Dr. Scott Harper at the Center for Gene Therapy at Nationwide Children’s. There, I focused on developing innovative gene therapy treatment approaches for Facioscapulohumeral muscular dystrophy (FSHD).
Joining Dr. Harper’s lab was one of the best decisions I have ever made. With the exceptional support of Dr. Harper and his team, I gained invaluable expertise in translational research, which enabled me to secure a research assistant professor position at the same center. With this new role, I was able to independently pursue my research vision and further contribute to the advancement in gene therapy for rare genetic diseases.
Why did you decide to pursue your work at Nationwide Children’s?
Nationwide Children’s is one of the best institutions for conducting translational gene therapy research, with two of the many FDA-approved gene therapies originating from its groundbreaking work. Several factors drew me to pursue my career at Nationwide Children’s. The institution actively encourages researchers to seek funding from diverse sources, including the NIH, foundations, and philanthropic organizations. Nationwide Children’s stellar reputation and collaborative culture also create valuable opportunities to build strong relationships with patient foundations and industry partners.
Additionally, Nationwide Children’s provides extensive support to secure funding and translate innovative ideas into actionable therapies. By being deeply invested in the success of its faculty and trainees, Nationwide Children’s creates a supportive ecosystem that has been instrumental in advancing my scientific goals while contributing to the development of life-changing therapies.
Fun Facts About Dr. Saad
What fictional character would you most like to meet and why?
The Giver (from The Giver by Lois Lowry)
I would love to ask him about the burden of holding all of humanity’s memories and history, and what wisdom he would share about free will, emotions, and the importance of passing humanity’s history to future generations.
What’s your favorite food?
I love Mediterranean foods, like tabouleh. But also, steak and fries.
What is your favorite band/genre/artist?
I enjoy a wide variety of music genres, but Rock is my favorite.
What is your favorite way to relax?
When the weather is nice, I enjoy going for a walk to unwind. I also appreciate the chance to relax and take a quick nap on the couch.
How did you meet your wife?
We met in France while attending the University of Strasbourg.
What’s next? What do you hope to accomplish in your research and professional development going forward?
My lab’s vision is to see novel and effective gene therapy solutions transform the outcomes for patients with genetic diseases, improving their quality of life. Our mission is to understand the causes of genetic disorders, including neuromuscular diseases (NMDs), and develop innovative approaches to diagnose and treat these conditions. The diseases under investigation include Facioscapulohumeral muscular dystrophy (FSHD), Duchenne muscular dystrophy (DMD), and laminopathies (i.e., progeria).
We will accomplish this mission by focusing on the translation of novel viral (adeno-associated virus) and non-viral (extracellular vesicle)-based gene therapy approaches to the clinic and extracellular vesicle biomarker discovery for genetic diseases. In addition, we will push to develop novel delivery approaches and platforms to enhance tissue specificity, efficacy and potency of viral and non-viral gene therapies. One of my goals is to cultivate strong partnerships with stakeholders to ensure that our research translates into safe and effective therapies.
On the professional development level, I am dedicated to further strengthening my leadership, project, and team management skills by staying up to date on best practices that enhance my team’s ability to be innovative, productive and fulfilled.
