Pediatrics
Showing 1-20 of 36 Results
-
Pasqualina Colella
Sr Res Scientist-Basic Life, Pediatrics - Genetics
BioDr. Pasqualina Colella is a scientist with extensive expertise in preclinical gene and cell therapy for rare inherited diseases.
She is currently a Senior Research Scientist at Stanford University, where she is developing innovative cell therapy approaches to address neuropathic lysosomal storage diseases (LSDs) using hematopoietic stem cell transplantation (HSCT) and genome editing. Her particular focus is on untreatable diseases caused by progranulin deficiency.
During her PhD research at TIGEM (IT), she developed therapeutic approaches for inherited blindness caused by the degeneration of retinal photoreceptors and dual AAV-mediated large gene delivery to the retina for USHIB and STGD.
During her postdoctoral studies at Genethon (FR), Dr. Colella focused on gene therapy for diseases caused by lysosomal enzyme deficiencies, particularly Pompe disease. She addressed immune and efficacy limitations of preclinical in vivo AAV gene therapy through transgene engineering, including secretable GAA and tandem promoters, while also investigating lysosomal enzyme trafficking and immune responses to gene therapy.
Dr. Colella has a proven track record, with 30 published manuscripts (16 as first author, 6 as corresponding author) and 5 patents.
She is dedicated to driving innovations in gene and cell therapy for untreatable diseases and to mentoring the next generation of passionate and rigorous gene and cell therapists.