Principal InvestigatorSeattle Children's Research InstituteSeattle, Washington, United States
PB0184 - Ultrasound Mediated Delivery of CRISPR/Cas9 Plasmid in Hemophilia A Mice Shows Therapeutic levels of FVIII Following NHEJ Gene Editing
Sunday, June 25, 202318:30 – 19:30 ET
OC 30.1 - A Novel Strategy for Platelet-Specific Gene Therapy for the Treatment of Hemophilia A via Intranasal Delivery Lentiviral Vectors Containing Factor VIII
Monday, June 26, 202310:45 – 11:00 ET
OC 30.3 - In vivo gene editing of hemophilia A mice using CRISPR/Cas9 mRNA LNP shows long-term expression of endogenous FVIII
Monday, June 26, 202311:15 – 11:30 ET