Our goal is that, through the multidisciplinary collaboration among the scientists of the MAGIC consortium, we will be able to effectively screen and validate new neuromuscular gene therapy vectors and genome-editing strategies, with high efficacy, low toxicity, and limited immunogenicity.

Each month, we gather partners from the MAGIC project to discuss aspects in relation to gene therapies. Readers will learn who the people behind the MAGIC Project are, why we are committed to advancing gene therapies, and how our roles in MAGIC are crucial for achieving better health outcomes for people living with muscular dystrophies. This time, we are speaking with Paola Galbiati from Gene Editing team at Genethon Institute.

In brief words, please let us know who you are individually and on behalf of which project partner organization. 

I am Paola Galbiati, a PhD student in Dr. Mario Amendola’s “Therapeutic Genome Editing” team at the Genethon Institute, located within the Genopole campus in Èvry. Genethon is a non-profit organization founded by the AFM-Telethon in 1990. As part of the Biotherapies Institute for Rare Diseases, its mission is to develop gene therapy treatments for rare genetic disorders and transition them to clinical application. The Therapeutic Genome Editing team focuses on translational research in gene therapy and genome editing, leveraging innovative technologies to design novel therapeutic strategies.

Why are you participating in the MAGIC project? How can your perspectives complement the MAGIC project goal to accelerate the development of genetic therapies for muscular dystrophies? 

As part of the MAGIC project, we are co-leading the consortium alongside Prof. Tedesco. Our work will focus on developing new treatments for neuromuscular disorders, specifically on Duchenne Muscular Dystrophy (DMD). Our research team specializes in CRISPR/Cas9, a groundbreaking technology that helps us modify genes in precise ways, such as correcting or replacing damaged genes. This technology holds great potential for both understanding and treating diseases. We are dedicated to creating new CRISPR/Cas9-based strategies that could benefit patients with DMD. These approaches will be tested in collaboration with other experts to ensure they are safe and effective, using laboratory models that closely mimic human conditions. Additionally, our team will support others in designing strategies for different types of muscular dystrophies and help assess the safety and accuracy of these treatments through specialized testing methods we have developed.

What have been the current challenges regarding the development of genetic therapies for muscular dystrophies for you? 

Significant progress has been made in understanding and treating DMD, one of the most common types of muscular dystrophy. Recently, the first gene therapy for DMD was approved. This therapy uses a viral vector to deliver a shortened version of the missing dystrophin gene (micro-dystrophin), which helps the body produce a protein similar to the one found in healthy muscle cells. Several other potential therapies that aim to restore dystrophin are currently being tested, with promising results in animal studies. However, when moving to human clinical trials, these treatments may face challenges, such as limitations in their effectiveness and potential immune system risks. To address these challenges, there is a strong need for improved models of human skeletal muscle disorders to test these therapies. These models can provide valuable insights into how well gene therapies might work in people and help predict the outcomes for patients.

What are the main outcomes (direct results) you expect from the project? 

Our goal is that, through the multidisciplinary collaboration among the scientists of the MAGIC consortium, we will be able to effectively screen and validate new neuromuscular gene therapy vectors and genome-editing strategies, with high efficacy, low toxicity, and limited immunogenicity.

What are the expected impacts on your organisation from participating in the project?

Participating in the MAGIC consortium gives our organization the opportunity to collaborate with a group of experts from different fields. This collaboration allows us to exchange knowledge and innovative ideas, enabling us to develop and test innovative therapies and technologies applicable to the neuromuscular field. By working closely with such a diverse and skilled group of scientists, we can enhance our research capabilities, refine our approaches, and accelerate the translation of new ideas into practical applications. This project aligns perfectly with the mission of our hosting institute, Genethon, and strengthens our ability to deliver impactful solutions for the patients.