Marcella GARITA-HERNANDEZ
Head of iPS Core Harvard Medical School
Dr. Marcela Garita-Hernandez, PharmD., PhD is a cell and gene therapist for inherited retinal diseases (IRDs) with a strong research background in stem cell biology and retinal differentiation. She has worked with stem cells from different sources since 2005 and evolved professionally along with the field of retinal differentiation from 2D adherent cultures to self-organizing 3D micro physiological systems such as the retinal organoid model. She pursued her PhD in cell therapy and regenerative medicine under the supervision of Shomi Bhattacharya at the Andalusian Center of Molecular Biology and Regenerative Medicine in Seville, Spain, followed by postdoctoral stages at the Institute de la Vision in Paris and the Institute des Neurosciences de Montpellier in France. Her primary objective is to unravel the mechanisms underlying photoreceptor cell death and retinal degeneration, with the ultimate goal of advancing novel genetic therapies. In 2021, she became an emergent group leader at the Ocular Genomics Institute (OGI), dedicating her research efforts to the development and characterization of human disease models of IRDs. Her science combines pluripotent stem cell technologies, genome editing, optogenetics, and other gene therapy tools to modify retinal organoids and retinal pigment epithelial cells of human origin. Marcela is the director of the iPS Research program and a group leader at the OGI, directed by Eric A. Pierce
Seminars
- Field overview of patient-derived iPSCs and retinal organoids, which provide a disease-relevant, human preclinical platform enabling functional validation of gene editing and cell therapies directly in photoreceptors, overcoming limitations of animal models
- Functional recovery validates therapeutic potential: restoration of key phototransduction components (e.g., PDE6, RHO, and others) in organoids confirms that genetic correction translates into biologically meaningful rescue, supporting singledose, gene and mutation-targeted therapies
- This approach helps to evaluate the efficacy, delivery strategies, and safety of genome-editing therapies across diverse monogenic diseases, supporting both regulatory decision-making and therapeutic development pipelines
