Quest for the cure: towards improving hematopoietic stem cell based lentiviral gene therapy

Abstract

In this thesis, we aim to tackle several key challenges in HSC-based gene therapy. Our primary objectives include the development of culture protocols for the ex vivo expansion of HSCs and the improvement of LV transduction efficiency in HSCs to facilitate gene therapy applications. Additionally, we seek to create a novel packaging cell line for LV production to reduce the high costs associated with gene therapy product manufacturing.