
A single systemic dose of an immune-shielded lentiviral vector delivering the MMUT gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia, according to newly published preclinical data.
Expert hematologist/oncologist Paul Szabolcs, MD, reviews positive data from a clinical trial comparing hematopoietic stem cell transplantation and omidubicel with standard cord blood transplantation.
Paul Szabolcs, MD, discusses data from the following study:






