School of Medicine
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Pasqualina Colella
Senior Research Scientist, Pediatrics - Genetics
BioDr. Pasqualina Colella is a distinguished scientist with expertise in gene and cell therapy for inherited diseases. She is currently a Senior Research Scientist at Stanford University, where she leads innovative translational research on hematopoietic stem cell transplantation (HSCT), microglia replacement strategies, myeloid cell recruitment to the central nervous system, and genome editing, with the ultimate goal of improving treatment outcomes for neurodegenerative lysosomal storage diseases.
Dr. Colella earned her PhD at TIGEM (Italy), where she developed gene therapies for inherited retinal disorders, including dual AAV approaches for large gene delivery targeting Usher syndrome type 1B and Stargardt disease. During her postdoctoral studies at Genethon (France), she advanced AAV-based gene therapy for Pompe disease, improving efficacy through transgene and promoter engineering. Her work has supported several ongoing clinical trials, including those for USH1B and Pompe disease.
Her research has resulted in over 30 peer-reviewed publications (16 as first author, 7 as co-corresponding) and 6 patents. She is the recipient of prestigious awards such as the Marie Skłodowska-Curie Fellowship and Innovative Research Grants from the National MPS Society.
