Introducing Lena Heer DC10

My PhD goals: The goal of my PhD project is to develop gene-based therapeutic approaches for cystinosis, a rare genetic disorder, using advanced organoid-based models. Gene editing via CRISPR-Cas9 will be used to establish a kidney organoid model of cystinosis derived from induced pluripotent stem cells (iPSCs). The aim is to restore gene function in these 3D models and to improve gene therapy delivery, which is currently a bottleneck for genetic kidney diseases.

My Background: I completed my BSc in Biotechnology at the University of Applied Sciences in Esslingen, Germany, with a strong emphasis on practical experience. During an internship at Novartis Pharma AG in Basel, Switzerland, I worked on developing viral vectors for gene therapy. For my BSc thesis, I joined Roche in Zurich, Switzerland, where my project focused on creating a molecular vector toolbox to accelerate the drug development process. I pursued my MSc in Pharmaceutical Design and Engineering at the Technical University of Denmark (DTU). My MSc thesis, conducted at DTU Health Technology under Prof. Sine Reker Hadrup, involved developing next-generation Chimeric Antigen Receptor (CAR)-T cells.

My research interests: My research interests lie in the field of cell and gene therapy. I particularly enjoy working on the drug development process, aiming to provide solutions that can improve the quality of life for people with specific diseases. Additionally, I strive to contribute to the development of relevant in vitro disease models to reduce reliance on animal studies in preclinical research, addressing ethical concerns and better recapitulating human diseases.

My hobbies: In my free time, I enjoy staying active with outdoor activities such as hiking, running, and climbing. When indoors, I love reading and experimenting with new cooking recipes.

Master thesis: Screening and delivery of recombinant proteins for next-generation CAR-T cell development in cancer immunotherapy.

CAR-T cell therapy shows promise in treating cancers, especially haematological malignancies, but faces challenges such as low efficacy in solid tumours, severe side effects, and immune escape of cancer cells. Next-generation CAR-T cell therapies are being developed to address these issues. This research introduces tools for engineering CAR-T cells to deliver recombinant proteins via the granzyme-perforin pathway, enabling precise cell-to-cell delivery. Flow cytometry-based assays and a pH-sensitive reporter have been developed to track protein localization and delivery, opening up potential applications for enhanced cancer cell killing.