Marcela Garita Lab
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Intro
The Garita Lab focuses on disease modeling and therapeutic development for inherited retinal disorders (IRDs) and other complex retinal diseases. Established in 2021, the lab has developed and characterized human retinal degeneration models to investigate disease mechanisms and support therapeutic discovery, including pharmacotherapy, gene and genome therapies, biomarkers discovery, and optogenetics.
Our mission is to advance understanding of the molecular basis of inherited retinal degeneration and to develop genetically informed therapies for vision loss.
The Garita Lab has active research projects regarding:
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We integrate hiPSC derived systems for various applications. We derived hiPSC via reprogramming from patients, and unaffected family members or lately we have also been using genome engineering to generate isogenic models of disease
- NAM Technologies
- Derivation of hiPSC cell lines
- To introduce mutations with precision we have optimized prime editing into hiPSC
- Optimization of prime editing in hiPSC to generate isogenic models of disease
- Generation of physiologically relevant human retinal organoids
- Human retinal organoid atlas
- Retinal Reporter lines
- Disease Modeling and therapeutic development for RNA splicing factor associated retinitis pigmentosa
- Disease Modeling and therapeutic development for NMNAT1 Leber Congenital Amaurosis (LCA)
- Disease Modeling and therapeutic development for Complex Retinal Dystrophies
- AMD
- Diabetic retinopathy
- Disease Modeling of Microphthalmia
- Genome editing for autosomal dominant IRDs
- PRPF8
- Gene Therapy validation
- PRPF31
- Elusive Gene Project
iPS Research Program
Director Dr. Marcela Garita-Hernandez, PharmD., PhD.
The iPS Research program is an interdisciplinary research program that offers training on basic and advance stem cell biology techniques on collaborative basis.
Four Research modules are covered in a 2-year program seminar series
- Retinogenesis
- Disease Modeling
- Transcriptomics
- Genome 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
- Retinal Ganglion Cell generation
- Disease modeling and phenotypic characterization
Current members in training:
- Egle
Past members
- Heran Getachew
- Hanmeng Zhang
- Michael Scandura
- Chris Miller
- Mohammed Zaeiter
- Hafiz Baig
- Rodrigo Cerna
Collaborations
- Eric Pierce
- Jason Comander
- Kinga Bujakowska
Stem Cell Medicine Group
Group Leaders Marcela Garita-Hernandez
The stem cell medicine group is a translational research group that aims for the better understanding of the mechanisms of human retinal pathogenesis with the perspective of developing new therapies. We use hiPSC-derived retinal tissues such as retinal organoids and RPE cells carrying mutation- causing diseases to model IRDs. The translational research of our group spans from diagnoses to modeling of IRDs for validation of new therapies in a human context
Projects
- Biomarker identification to enable accurate earlier diagnosis of IRDs
- Disease Modeling of inherited developmental and adult retinal disorders
- Gene therapy tools validation
- Drug discovery
Publications
Scientific Papers
For extended list and full information visit my research gate profile.
- Sanjurjo-Soriano, C., Erkilic, N., Damodar, K. Hassan Boukhaddaoui, H., Diakatou, M., Garita-Hernandez, M., Mamaeva, D., Dubois, G., Jazouli, Z., Jimenez-Medina, C., Goureau, O., Meunier I., Kalatzis, V. Retinoic acid delays initial photoreceptor differentiation and results in a highly structured mature retinal organoid. Stem Cell Res Ther 13, 478 (2022). doi:10.1186/s13287-022-03146-x
- Garita-Hernandez, M., Chaffiol, A., Guibbal, L., Routet, F., Khabou, H., Toualbi, L., Riancho L., J., Sahel, J.-A., Duebel, Goureau, O., Dalkara, D. Control of microbial opsin expression in stem cell derived cones for improved outcomes in cell therapy. Frontiers in Cellular Neurosciences (2021) # Corresponding Author
- Garita-Hernandez, M., Routet, F., Guibbal, L., Khabou, H., Toualbi, L., Riancho L., Reichman, S., Duebel, J., Sahel, J.-A., Goureau, O., Dalkara, D. AAV-mediated gene delivery to 3D retinal organoids derived from human induced pluripotent stem cells. International Journal of Molecular Sciences (2020) 21(3):994 doi:10.3390/ijms21030994
- Garita-Hernandez, M*., Lampic, M*., Chaffiol, A., Guibbal, L., Routet, F., Santos-Ferreira, T., Gagliardi, G., Reichman, S., Picaud, S., Sahel, J.-A., Goureau, O., Dalkara, D., Duebel, J. Restoration of visual function by transplantation of optogenetically engineered photoreceptors. Nature Communications (2019) 10, 4524 doi:10.1038/s41467-019-12330-2
- Garita-Hernandez, M#., Guibbal, L., Toualbi, L., Routet, F., Chaffiol, A., Winckler, C., Harinquet, M., Robert, C., Fouquet, S., Bellow, S., Sahel, J.A., Goureau, O., Duebel, J., Dalkara, D., Optogenetic Light Sensors in Human Retinal Organoids. Frontiers in Neurosciences (2018) 12, 789. doi:10.3389/fnins.2018.00789 # Corresponding Author
- Khabou, H., Garita-Hernandez, M., Chaffiol, A., Reichman, S., Jaillard, C., Brazhnikova, E., Bertin, S., Fradot, V., Desrosiers, M., Winckler, C., Goureau, O., Picaud, S., Duebel, J., Sahel, JA, and Dalkara, D. Noninvasive gene delivery to foveal cones for vision restoration. JCI Insight (2018) 3. doi:10.1172/jci.insight.96029
- 2018 International Patent (WO/2018/055131, submitted by Sorbonne University, INSERM and CNRS) as an Inventor. Title: ‘Optogenetically transformed photoreceptor cells for use in the treatment of retinal degenerative diseases’. Partner Inventor.
- Garita-Hernandez M, Goureau O, Dalkara D. Gene and Cell Therapy for Inherited Retinal Dystrophies. eLS. (2016). 1–16. doi: 10.1002/9780470015902.a0026565
- Garita-Hernández M., Diaz-Corrales F., Lukovic D., Gonzalez-Guede I., Díez-Lloret A., Sánchez-Valdés ML., Massalini S., Erceg S., Bhattacharya SS. Hypoxia Increases the Yield of Photoreceptors Differentiating from Mouse Embryonic Stem Cells and Improves the Modeling of Retinogenesis in vitro. Stem Cells (2013) 31(5):966-78. doi:10.1002/stem.1339. PMID: 23362204
- Erceg, S; Moreno-Manzano, V; Garita-Hernandez, M; Stojkovic, M; Bhattacharya, SS; Concise review: stem cells for the treatment of cerebellar-related disorders. Stem Cells (2011) 29 (4):564-569 doi:10.1002/stem.619
Theses
Garita-Hernández M., Influence of oxygen tension on the differentiation of embryonic stem cells towards photoreceptors and other retinal phenotypes (2013) CSIC-JA-UPO-USE-Centro Andaluz de Biología Molecular y Medicina Regenerativa, Thesis Disertation
Patents
For full details about my patents, please browse WIPO website.
- 2017 European Patent (EP3516046, n°16 306 225): ‘Optogenetically transformed photoreceptor cells for use in the treatment of retinal degenerative diseases’. Partner Inventor
- 2018 International Patent (WO/2018/055131): ‘Optogenetically transformed photoreceptor cells for use in the treatment of retinal degenerative diseases’. Partner Inventor
Software
Grants
- Knights Templar Eye Foundation Career-Starter Research Grants (2022-2023)
- Knights Templar Eye Foundation Competitive Renewal Grants (2023-2024)
Funding
Work Experience
2021 – current position | Ocular Genomics Institute, Mass Eye and Ear, Harvard Medical School.
Director of the iPS Research Lab at the Ocular Genomics Institute of the Mass Eye and Ear.
Appointed Instructor of Ophthalmology, Harvard Medical School
TODO: complete with field, Projet, and responsabilities info.
2019 – 2021 | Institut des Neurosciences de Montpellier, France
Posdoctoral Researcher | Supervisor: Dr. Vasiliki Kalatzis
Gene Therapy of inherited retinal diseases due to large causative genes
Project
- Validation of a large capacity vector for retinal delivery of genes with large cDNA sequences
- Viral-mediated gene delivery in human photoreceptors and RPE cells derived from hiPSCs
Personnel under my supervision:
- Carla JIMENEZ, MSc student Erasmus plus fellowship
2015 – 2019 | Institut de la Vision – Paris, France
Postdoctoral Researcher | Supervisor: Dr. Deniz Dalkara
Genetic engineering of retinal organoids derived from human induced pluripotent stem cells
Projects
- Generation and characterization of retinal organoids derived from hiPSCs
- Maturation of photoreceptors derived from hiPSCs
- Viral-mediated expression of rod-derived cone viability factor to ameliorate the cones phenotype
- Transplantation of optogenetically-modified photoreceptors derived from hiPSCs in models of retinal degeneration
- Safety profiling of optogenetic tools in retinal organoids derived from hiPSCs
Patents
- 2017 European Patent (EP3516046, n°16 306 225): ‘Optogenetically transformed photoreceptor cells for use in the treatment of retinal degenerative diseases’. Partner Inventor
- 2018 International Patent (WO/2018/055131): ‘Optogenetically transformed photoreceptor cells for use in the treatment of retinal degenerative diseases’. Partner Inventor
Personnel under my supervision:
- Research Assistants
- Laure GUIBBAL, MSc 3 years
- Fiona ROUTET, MSc 3 years
- Lyes TOUALBI, MSc 1 year
- Interns | Bachelor degree
- Karen DAOUD, Sorbonne University. 1.5 months 2015
- Marilyn HARINQUET, Evry University. 2 months 2015
- Interns | Master degree
- Karen DAOUD, Université Paris Descartes. 10.5 months 2017
- Marilyn HARINQUET, Sorbonne University. 4 months 2016
- Fiona ROUTET, Sorbonne University. 6 months 2016
- Parisa KHALILIAN, Sorbonne University. 5 months 2017
- Lyes TOUALBI, Université Paris Saclay. 8 months 2017-18
2009 – 2013 | CABIMER – Seville, Spain
PhD Student and Research Assistant | Supervisor: Dr. Shomi Bhattacharya.
Retinal Degeneration lab, Cell therapy and Regenerative Medicine, Cabimer.
Personnel under my supervision (Research Assitants):
- Andrea Diez Lloret, PhD
- Simone Massalini, MSc
2008 – 2009 | CABIMER – Seville, Spain
Research Assistant | Supervisors: Bernat Soria and Karim Abdelcrim.
Laboratory of Stem Cells.
2005 – 2007 | Instituto de Biomedicina de Sevilla, IBIS – Seville, Spain
MSc. Fellowship | Supervisor Dr. Juan José Toledo-Aral.
Evaluation of the neuroprotective effect of the antioxidant drug NP061017 using the acute Parkinsonism murine model of MPTP
2004 – 2005 | Vivacell Biotechnology/Universidad de Córdoba – Spain
Research Assistant | Supervisor: Dr. Eduardo Muñoz Blanco.
Laboratory of Immunology, Physiology and Cell Biology.
Cytotoxicity Assays in different cell lines. Determination of the biological activity of Cimicifuga racemosa extract.
2002 – 2004 | Universidad de Costa Rica – San José, Costa Rica
Lecturer of Toxicology and Pharmacology, Faculty of Pharmacy
Researcher Centre of Research in Neurosciences | Supervisor: Dr. Jaime Fornaguera Trías.
Teaching
Invited lecturer:
- Master 2 in Neurosciences, Ecole Pratique de Hautes Etudes,
Montpellier, France - International Master Programme on Visual Science, UNAM,
Sorbonne Université. Paris, France - International Master on Stem Cell Biology, Sorbonne Université.
Paris, France - Distinguished Academic Researcher, Faculty of Farmacy,
Universidad de Costa Rica
Reviewing Activities
- Editorial Board, Frontiers in Molecular Neuroscience journal,
- Scientific Advisory Board,
Center of Research in Neuroscience / University of Costa Rica / Costa Rica, - International peer reviewer for research projects,
University of Costa Rica, Costa Rica, - Reviewer, Stem cells, Plos Biology, International Journal of Molecular Sciences and Trends in biotechnology journals.
Software
Mageek
Mageek is an ImageJ Plugin to colorize microscope Images in batch processing.
By default, images are in level of grey, Mageek colorize them using LUT presets. You can handle hundred of files in batch processing.
Each of those extensions correspond to different microscope image file format.
Links:
Examples:
TODO: add media here to showcase various Mageek input-outputs.
Authors:
Marcela Garita H (Science).
Bérenger Dalle-Cort (Code, UI & art)
Frequently Asked Questions
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This exquisite compilation showcases a diverse array of photographs that capture the essence of different eras and cultures, reflecting the unique styles and perspectives of each artist.
Are signed copies available?
This exquisite compilation showcases a diverse array of photographs that capture the essence of different eras and cultures, reflecting the unique styles and perspectives of each artist.
Contact
Marcela Garita-Hernández, PhD, PharmD
Retinal Development, Cell and Gene Therapies
Senior Research Fellow at Mass Eye and Ear, Harvard Medical School
Boston, Massachusetts, USA.
ORCID 0000-0002-4311-138X
