A glimpse into SR-Tiget research at ASGCT 2024!
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)
Performing innovative gene and cell therapy research and clinical trials with a main focus on genetic diseases
Invited speakers
May 7, 8:25 am - Samuele Ferrari (Naldini’s lab): “Leveraging on the LNP/mRNA technology for ex-vivo gene editing”;
May 9, 09:18 am - Alessandro Aiuti : “Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders”
May 11, 8.15 am - Michela Milani (Cantore’s lab): “Possible immune responses to in vivo delivery approaches for hematologic disorders and ways to overcome them”
May 11, 08:50 am?- Luigi Naldini: “Comparing and Contrasting Viral and Non-Viral Delivery”
Abstracts selected for oral presentation
May 7, 1.45 pm - Maria Ester Bernardo : “Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-Correction Mechanisms”
May 8, 11:15 am - Alessandro Aiuti : “Atidarsagene Autotemcel (Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-Up”
May 9, 9:15 am - Monica Volpin (Montini’s lab):?? “Harnessing Chromatin Architecture and Post-Transcriptional Regulation to Benefit the Safety of the Lentiviral Vector Platform”
May 9, 1:30 pm - Elena Barbon (Cantore’s lab): “Long-Term Efficacy, Safety, and Extrahepatic Benefit of Liver-Directed Lentiviral Gene Therapy in a Methylmalonic Acidemia Mouse Model”
May 9, 3:45 pm - Cesare Canepari (Cantore’s lab): “Lentiviral Gene Therapy to the Liver for Homozygous Familial Hypercholesterolemia (FH): Assessing the Best Vector Design”
May 10, 1:45 pm - Daniele Canarutto (Naldini’s lab): “Heterogeneous On-Target Genomic Rearrangements Occur upon Long Range Gene Editing in T-Cells”
领英推荐
May 10, 5:00 pm - Carlo Cipriani, (Cesana/Montini’s lab): “Characterization of AAV Integrations in Preclinical Models of Gene Therapy Using RAAVioli Pipeline with Long and Short Sequencing Reads”
May 11, 10:30 am - Cristina Colleoni (Montini’s lab): “A Genotoxicity Mouse Model of HSPC Gene Therapy to Identify Early Biomarkers in the Interaction between Senescent Cells and the Immune System”
May 11, 11:45 am - Francesco Gazzo (Montini‘s lab): “Somatic Mutation Tracking in Hematopoietic Stem Cell Gene Therapy Reveals Absence of Clonal Hematopoiesis”
Posters
May 8 - Fabrizio Benedicenti (Montini’s lab): “Optimization of Sonication Linker Mediated (SLiM)-PCR for Vector Integration Sites Retrieval and Quantification”
May 9 - Chiara Simoni (Cantore’s lab): “Assessing the Best Strategy and Timeframe for In Vivo Genome Editing of Progressive Familiar Intrahepatic Cholestasis Type 2”?????????????????????????????????????????
May 10??- Samuele Ferrari (Naldini’s lab): “Efficient Ex-Vivo Selection of Gene Edited Hematopoietic Stem Cells by Leveraging on Artificial Transactivators and Lipid Nanoparticle-Mediated Delivery”???????
May 10??- Samuele Ferrari (Naldini’s lab): “Targeted Base Editing Models Hematopoiesis and Pathophysiology of VEXAS Syndrome”
May 10 - Maria Ester Bernardo: “Non-Neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene Therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation”??????????????????????
May 10 - Maria Ester Bernardo: “Interim Analysis on Neurological Outcomes in Hurler Syndrome Patients Treated with Autologous Ex Vivo Hematopoietic Stem Cell Gene Therapy”
May 10 - Maria Ester Bernardo: “Pre-Clinical Development of an Ex-Vivo Lentiviral-Based Hematopoietic Stem/Progenitor Cells (HSPC)-Gene Therapy (GT) for Mucopolysaccharidosis Type IVA (MPSIVA) as Part of an Innovative GT Platform Approach for LSDs with Skeletal Involvement”
May 10 - Maria Ester Bernardo: “Lentiviral Vector Optimization for the Preclinical Development of Hematopoietic Stem Progenitor Cells (HSPCs)-Gene Therapy(GT) for Mucopolysaccharidosis Type IVB (MPSIVB)”