Master’s Thesis in Cardiac Gene Therapy and Translational Genome Editing

21.01.2026, Abschlussarbeiten, Bachelor- und Masterarbeiten

Cardiovascular diseases remain the leading cause of mortality worldwide, and many inherited and acquired cardiac disorders still lack curative treatment options. Recent advances in gene therapy and genome editing, particularly AAV-mediated CRISPR and base/prime editing technologies, have created new opportunities for precise and durable therapeutic interventions in the heart.
However, efficient in vivo genome editing of cardiomyocytes remains challenging. Cardiomyocytes are terminally differentiated, largely post-mitotic cells with limited DNA repair capacity, and therapeutic outcomes are strongly influenced by vector design, delivery route, developmental stage, and tissue-specific expression. Bridging the gap between small-animal models and clinically relevant large-animal systems, such as porcine models, is therefore a critical step toward translational cardiac gene therapy.

Our Approach
Our research focuses on the development and optimization of AAV-based gene therapy and genome editing platforms for cardiac applications. We combine:
• Advanced AAV vector engineering, including dual-AAV systems
• CRISPR- and base/prime-editing strategies tailored for cardiac tissue
• In vivo cardiac delivery in mouse and porcine models
• Molecular, histological, and functional assessment of editing outcomes
This integrated strategy enables mechanistic insight while supporting translational development toward clinical cardiac gene therapy.

Thesis Project Overview
Project Aim
The aim of this project is to systematically evaluate how delivery strategy, developmental stage, and model system influence genome-editing efficiency and therapeutic potential in the heart. By optimizing these parameters across species, the project seeks to enable robust, scalable, and translational cardiac genome-editing approaches.

Project Scope
The exact project will be tailored to the student’s background and current laboratory priorities. Possible components include:
• AAV-mediated gene delivery to the heart
• In vivo CRISPR or prime-editing experiments
• Comparison of neonatal, juvenile, and adult cardiac editing
• Molecular analysis of editing efficiency (PCR, qPCR, sequencing)
• Tissue distribution and expression analysis
• Contribution to translational studies in porcine cardiac models

Your Role
As a Master’s thesis student, you will be fully embedded in an active translational research environment. You will:
• Perform molecular cloning and AAV-related experiments
• Work with mouse and/or porcine models of cardiac gene therapy
• Analyze genome-editing outcomes at DNA and RNA levels
• Interpret results in a physiological and translational context
• Contribute to figures, datasets, and scientific manuscripts

Expected start: As soon as possible
Duration: 6- 12 months

Candidate Profile
• Background in molecular biology, biotechnology, biomedicine, or life sciences
• Strong interest in gene therapy, genome editing, or cardiovascular research
• Basic laboratory experience in molecular biology techniques
• Willingness to work with animal models (training provided)
• Prior experience with AAV, CRISPR, sequencing, or histology is an advantage but not required
What We Offer
• Hands-on training in state-of-the-art cardiac gene therapy and genome editing
• Exposure to both small- and large-animal translational research
• Close supervision and mentoring
• Opportunity to contribute to high-impact scientific publications
• A collaborative and international research environment

Supervision
This master’s thesis will be conducted in the research group of Prof. Dr. med. Christian Kupatt, with close supervision by Dr. Vijayanand Rajendran and Dr. Tarik Bozoglu

How to Apply
Please email your CV and a brief introductory email outlining your background and availability.

Contact
Dr. Vijayanand Rajendran (vijayanand.rajendran@tum.de)
Klinik und Poliklinik für Innere Medizin I – Kardiologie
TUM Klinikum Rechts der Isar
Ismaninger Straße 22, 81675 München

Kontakt: vijayanand.rajendran@tum.de