Antonia Follenzi, MD, PhD
Highlights From NHF's 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia Targeting Sinusoial Endothelium Antonia Follenzi, MD, PhD University of Piemonte Orientale Novara, Italy Key Data Points pF8 Promoter Drives GFP Expression in LSECs LV-pF8-FVIII Corrected FVIII Level in B6/129-HA Mice is Treg Dependent The natural FVIII promoter, pF8, drives GFP expression in liver sinusoidal endothelial cells (LSECs). Immunofluorescence analysis of liver of C57BL/6 hemophilia A (HA) mice, 1 and 6 months after injection of the lentiviral vector LV.pF8.GFP. (DAPI, 4′,6-diamidino-2-phenylindole; GFP, green fluorescent protein; Lyve-1, lymphatic vessel endothelial receptor 1, a marker for LSECs) To summarize this series of studies, immunosuppression with minimal reduction in Treg cells is optimal for ensuring liver-mediated immune tolerance to FIX transgene expression. In NHPs, pretreatment with MMF and rapamycin, followed by delayed administration of rATG was able to provide sustained immune tolerance whereas rATG or daclizumab administered at the time of vector infusion was not. RELATED CONTENT Interactive Webinars Podcasts Gene Therapy as a Global Solution Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Johnny Mahlangu, BSc, MBBCh, MMed, FCPath Podcasts CREATED_BY Super User 06 December 2021 Gene Therapy for Hemophilia A and B Compared to Available Treatments Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Maria Elisa Mancuso, MD, PhD Podcasts CREATED_BY Super User 06 December 2021 Patient Perspectives for Hemophilia in Gene Therapy Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Declan Noone Podcasts CREATED_BY Super User 06 December 2021 Gene Therapy in Hemophilia Ongoing and Future Research and What to Expect Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Dr. Steven W. Pipe, MD Podcasts CREATED_BY Super User 06 December 2021 Preparing Treatment Centers for Administration Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Wolfgang A. Miesbach, MD, PhD Podcasts CREATED_BY Super User 06 December 2021 See All Podcasts
