Our recent manuscript shows that EVs from the PRPF31 mutant RPE cells contain polyadenylated RNAs and microRNAs that are linked to RPE de-differentiation and degeneration, highlighting their potential as circulating biomarkers for RP. This research paves the way for the development of non-invasive diagnostic tools that could transform disease monitoring and therapeutic assessment. These findings were published in the Molecular Therapy: Methods & Clinical Development journal https://lnkd.in/enCkEYNt
iPS Research Lab
Biotechnology Research
Boston, Massachusetts 64 followers
3D human retina models for developing new therapeutics
About us
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:
1. Biomarker identification to enable accurate earlier diagnosis of IRDs.
2. Disease Modeling of inherited developmental and adult retinal disorders.
3. Gene therapy tools validation.
4. Drug discovery
The iPS Research Lab has an interdisciplinary research program that offers training on basic and advanced stem cell biology techniques on a collaborative basis.
Four Research modules are covered in a 2-year program
1. Retinogenesis
2. Disease Modeling
3. Transcriptomics
4. Gene Editing
Hands-on training includes:
1. Gene editing of iPSC lines using canonical CRISPR-Cas9, prime editing or base editing technologies.
2. Cell banking and QC of iPSC lines
3. Genotyping and sequencing analysis
4. RPE differentiation
5. Retinal Organoids generation
6. Disease modeling and phenotypic characterization
- Website
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https://oculargenomics.meei.harvard.edu/team/marcela-garita-hernandez/
External link for iPS Research Lab
- Industry
- Biotechnology Research
- Company size
- 11-50 employees
- Headquarters
- Boston, Massachusetts
- Type
- Privately Held
- Specialties
- 3D retinal models, disease modeling, and genome editing
Locations
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Primary
Get directions
243 Charles Street
Ocular Genomics Institute
Boston, Massachusetts 02114, US
Updates
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Take a look at our latest article featuring a pipeline to safely generate isogenic models of disease using #primeediting and #hiPSC. Ocular Genomics Institute Massachusetts Eye and Ear Harvard Medical School
The genetic background of a model may contain additional variants that contribute to the disease and complicate phenotype rescue. In this manuscript, we utilize prime editing technology to safely introduce point mutations into hiPSCs and generate isogenic disease models. With our optimized system, we successfully address the low efficiency typically observed in hiPSCs. Learn more here: https://lnkd.in/efyuKP_i
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