iPS Research Lab at the Ocular Genomics Institute
Director: Dr. Marcela Garita-Hernandez, PharmD., PhD.
With the goal of developing new therapies, the iPS Research Laboratory is a translational research group that strives to understand the mechanisms underlying human retinal pathogenesis. Our approach involves utilizing patient-derived hiPSC (human-induced pluripotent stem cells) and isogenic model lines generated through cutting-edge gene editing technologies, such as base and prime editing.
Current Research
Dr. Garita-Hernandez’s vast expertise generating hiPSC-derived retinal tissues such as various retinal organoids and RPE cells is used in projects spanning from diagnoses to modeling of inherited retinal diseases (IRDs) for validation of new therapies in a human context.
Main research topics:
- Biomarker identification to enable accurate earlier diagnosis of IRDs.
- Disease Modeling of inherited developmental and adult retinal disorders.
- Gene therapy tools validation.
- Drug discovery
The iPS Research Lab has an interdisciplinary research program that offers training on basic and advance stem cell biology techniques on a collaborative basis.
Four Research modules are covered in a 2-year program
- Retinogenesis
- Disease Modeling
- Transcriptomics
- Gene Editing
Hands on training includes:
- Gene editing of iPSC lines using canonical CRISPR-Cas9, prime editing or base editing technologies.
- Cell banking and QC of iPSC lines
- Genotyping and sequencing analysis
- RPE differentiation
- Retinal Organoids generation
- Disease modeling and phenotypic characterization
Contact information: Email: mgarita@meei.harvard.edu