Specialization
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Translational research on retinal degenerative diseases using induced pluripotent stem (iPSC) cell technology.
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Non-clinical study design
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Development of cell-based therapies for retinal degenerative diseases.
Focus of research
Dr. Mitra Farnoodian is an Assistant Professor at Amsterdam UMC, University of Amsterdam. Her group aims to advance understanding of the molecular basis of inherited eye diseases and to identify novel therapeutic strategies, particularly for conditions that currently lack effective treatments. For over 14 years, Dr. Farnoodian has dedicated her research to studying retinal pigment epithelium (RPE) cell biology using induced pluripotent stem cell (iPSC)-derived RPE from both healthy donors and patients with retinal disorders.
Her lab is committed to pioneering therapies for retinal degenerative diseases through cutting-edge translational research. By investigating cellular metabolic regulation and dysfunction in ocular tissues, the lab seeks to identify novel pharmacological and stem cell-based therapies to prevent RPE cell death, with a clear path toward clinical application. Through collaborative, interdisciplinary efforts, Dr. Farnoodian’s group aims to transform mechanistic insights into clinically relevant therapies that preserve and restore vision.
Background
Dr. Mitra Farnoodian earned her Master’s in Medical Microbiology from the University of Wisconsin–Madison. She completed her Ph.D. in Clinical Investigation at the University of Wisconsin–Madison, studying mechanisms of RPE and choroidal dysfunction in AMD, work that provided new therapeutic strategies and established her foundation in translating mechanistic discoveries into therapeutic concepts.
As a postdoctoral researcher and scientist at the National Eye Institute (NEI/NIH), Dr. Farnoodian led R&D programs that generated significant basic and clinical findings. She extensively investigated RPE cell biology using iPSC-derived RPE from healthy and diseased individuals, focusing on how lipid metabolism dysregulation and lysosomal dysfunction in ocular tissues drive disease progression and reveal novel treatment targets for retinal degenerative diseases. Her published work on Stargardt disease pathogenesis emphasizes the need for further research into lipid metabolism and signaling in the retina and RPE to guide effective therapeutic strategies.
Additionally, she contributed as a key team member to the first-in-human Phase I/IIa clinical trial of autologous iPSC-derived RPE transplantation for dry AMD, working collaboratively across multiple disciplines to ensure translational and regulatory readiness. Dr. Farnoodian’s work has been recognized with several prestigious awards, including the NIH Director’s Award – STEM RPE Surgical Transplant Dream Team, the NEI Director’s Awards – STEM RPE Surgical Transplant Dream Team, and the Scientific Medical Innovation Award for RPE and Photoreceptor Cell Therapy, and the KTEF Career Starter Grant for developing a stem cell-based drug discovery platform targeting lipid-handling defects in Stargardt disease.