Home Department: Bioengineering
Mentor: Matthew Porteus (Pediatrics)

"Engineering a High-Capacity, High-Efficiency Genome-Editing Platform for ALD Therapy"

X-linked adrenoleukodystrophy (ALD) is a fatal genetic disorder caused by mutations in the ABCD1 gene, leading to toxic lipid accumulation and progressive neurological damage. Autologous gene-corrected hematopoietic stem cell (HSC) transplantation is a promising therapy but is limited by inefficient CRISPR/Cas9 homology directed repair (HDR) mediated integration of large gene cassettes. Griffin’s project will combine selectable knock-in strategies with dual-AAV vectors to enable efficient, precise insertion of large therapeutic payloads. Integration of a corrected ABCD1 gene with GFP and luciferase reporters will allow in vivo tracking of therapeutic cells, advancing gene therapy for ALD and potentially other genetic diseases.