-
Eric D. Adler, MD
Clinical Professor, Department of Medicine
Director, Strauss Center for Cardiomyopathy
Dr. Adler is a Principal Investigator in clinical trials for all stages of heart failure. He is involved in clinical research examining the use of gene therapy and stem cells for treating heart disease. His lab's focus is on how genetic mutations cause cardiomyopathy. His recent work has been the study of Danon disease, a rare devastating inherited cause of heart failure.
-
Shyamanga Borooah, MBBS, PhD
Physician-Scientist, Shiley Eye Institute
Dr. Borooah has specific expertise in the latest gene therapeutic approaches for ocular disease being the principal investigator on some of the first gene therapy, CRISPR and antisense oligonucleotide clinical trials for eye disease. He has received numerous awards including a Fulbright scholarship for developing translational treatments for eye disease.
-
Stephanie Cherqui, PhD
Professor, Department of Pediatrics
Director, Gene Therapy Initiative
Dr. Cherqui is recognized for her gene therapy work, specifically in genetic disorders using stem cells. Named as a top academic entrepreneur by BIOS, she's renowned for her contributions to cystinosis research. Currently, she's leading the first stem cell gene therapy clinical trial for cystinosis at UC San Diego.
-
Chamindra G. Laverty, M.D
Professor, Department of Neurosciences
Director, Multidisciplinary Neuromuscular Clinics
Dr. Laverty is a board-certified neurologist specializing in the management and treatment of neuromuscular diseases. She is the Investigator for many clinical trial research studies in various muscular dystrophies at the UC San Diego School of Medicine. Her special interest lies in advancing disease-modifying therapies, such as gene replacement therapy, cell therapy, and anti-sense oligonucleotides for her patients.
-
Marsala Martin, MD
Professor, Anesthesiology Research Lab
Dr. Marsala has been the Principal Investigator for numerous NIH-funded projects and has helped develop novel delivery techniques for cell and gene-based therapeutics in treatment of neurological disorders including ALS and FTD. He is renowned for his research in cell-replacement therapy for the treatment of spinal injury, focused on the development and characterization of the potency of spinally-grafted cell grafting.
-
Chris Mason, MD, PhD
Clinical Professor of Cell and Gene Therapy at University College London
Dr. Mason is a clinician-scientist with over 30 years of cell and gene therapy experience spanning R&D, clinical medicine, bioprocessing, and business. Dr. Mason was instrumental in founding the Alliance for Regenerative Medicine [ARM] and is a current Board Director. He's also the Senior Editor of ‘Cell and Gene Therapy Insights’ and ‘Regenerative Medicine’ journals.
-
Alysson Muotri, PhD
Professor, Department of Pediatrics
Co-Director, Gene Therapy Initiative and Stanford Stemcell Institute
Dr. Muotri leverages stem cells to study the human brain. His achievements include demonstrating the functionality of human stem cell-derived neurons in chimeric mouse brains and creating a global model for neurodevelopmental disorders using stem cells. His work earned him prestigious accolades, such as the NIH Director's New Innovator Award.
-
Farah Sheikh, PhD
Professor, Department of Medicine
Dr. Sheikh's gene therapy research includes pioneering human-relevant mouse models, uncovering new molecular targets, and designing a gene therapy treatment for arrhythmogenic cardiomyopathy, currently in a Phase 1/2 clinical trial with LEXEO Therapeutics Inc. She has launched successful gene therapy start-ups and maintains industry partnerships.
-
Mark H. Tuszynski, MD, PhD
Professor, Department of Medicine
Director, Center for Neural Repair
Dr. Tuszynski's research focuses on growth factors & stem cells influencing survival, plasticity, and regeneration in adult central nervous system. In 2001, he led the first clinical trial of gene therapy for a neurological disorder, testing nerve growth factor delivery in Alzheimer's patients. He has won over 20 research awards and authored 300 scientific medical publications.