Our lab is currently welcoming students, post-docs, and research assistants.
About the Gomez-Ospina Lab
The main focus of Dr. Gomez-Ospina’s lab is to develop therapies for patients with genetic diseases. We use genome-editing and stem cells to produce definitive treatments for childhood neurodegenerative diseases, many of which are lysosomal storage disorders.
​Ongoing translational projects include developing autologous transplantation of genome-edited hematopoietic stem cells and microglia replacement therapy for:
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Mucopolysaccharidosis type I
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Gaucher disease
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Krabbe disease
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Frontotemporal Dementia
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Friedreich's ataxia.
We are also pursuing basic science questions such as:
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Describing new genetic diseases
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Increasing the efficiency of genome editing tools
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Understanding microglia turnover in response to conditioning
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Establishing brain-specific conditioning regimens.