Gene Editing/Gene Therapies Oral Abstracts
Friday, May 31, 2024 03:45 PM - 04:45 PM
Room 202-204
Gene Editing / Gene Therapies
Abstract Presentations
- Durable and Dose-Dependent Correction of the Biochemical Phenotype in a Mouse Model of Guanidinoacetate Methyltransferase (GAMT) Creatine Deficiency Following Treatment with scAAV9.hGAMT, Robyn Taylor Binsfeld, Canada
- Whole-Body Biodistribution of AAV-Mediated Gene Expression In Mice Using Multi-Resolution 3D Cryoviztm Imaging, Madhusudhana Gargesha, United States
- Therapeutic genome editing for severe monogenic skin disorders, John Murray Topp Hunt, New Zealand
- Development of a GMP Manufacturing Process for Base Edited Hematopoietic Stem and Progenitor Cells (BE-HSPCs) for X-linked Chronic Granulomatous Disease (X-CGD), Hong Lei, United States
- Cas9-mediated precise and template-free gene editing of a muscular dystrophy founder mutation: From single editing and off-target analysis to double editing and clinical translation, Stefanie Müthel, Germany
- Programming Naïve Primary T Cells for Enhanced Immunotherapy, Ankur Singh, United States
