The aim of MAGIC is to accelerate the development of gene therapies and genome editing for muscular dystrophies by creating advanced humanized muscle models and innovative approaches. The goal is to improve the lives of people living with muscular dystrophies.

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

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

Prof. Karen English, I am an Immunologist by training and my research is focused on cell and gene therapies. I am based at Maynooth University and I am the workpage 6 (Preclinical Immunology) leader. 

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? 

The MAGIC project is an exciting project seeking to advance gene therapies for muscular dystrophies. The project is incredibly important to develop much needed gene therapy for muscular dystrophies. The immune response to viral vectors and the transgenes that they deliver is a major consideration for MAGIC. I have long been interested in the immune response to cell-based therapies and I bring expertise in transplantation immunology that can be applied to gene therapy. 

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

Adverse events in boys who have received AAV gene therapy has been associated with immune system activation. The immunogenicity of transgenes such as microdystrophin is a very significant challenge that needs to be addressed. In addition, activation of the immune system against the gene therapy means that repeat dosing of the gene therapy is complicated by the immune system. 

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

We expect to develop an optimized, scalable AAV production process for novel muscle-targeted AAV serotypes that aligns with the unique needs of a gene therapy for muscular dystrophy. By working closely with the other consortium partners, we aim to create manufacturing solutions that not only support this project but also establish a foundation for future gene therapy production. Additionally, we anticipate advancing innovative technologies that enhance production efficiency, quality, and yield-ultimately contributing to making therapies more accessible.

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

We are hoping to identify the cells of the immune system that respond early to viral vectors and vector-transgenes and to develop ways to calm this immune response to allow the gene therapy to do it’s job.