The objectives and approach of MAGIC are as follows:
Objectives
- Advanced Muscle Models: Create highly accurate humanized muscle models for studying muscular dystrophies.
- Precision Gene Therapies: Develop precise gene therapy and genome editing strategies tailored to treat muscular dystrophies.
- Muscle-on-Chip Devices: Engineer disease-specific muscle-on-chip devices using cutting-edge technologies.
- Gene Therapy Vector Engineering: Using advanced muscle models, design and evaluate safer, more efficient, and targeted novel gene therapy vectors for neuromuscular diseases.
- Gene Editing Strategies: Explore innovative gene editing strategies to address specific mutations in neuromuscular disorders, including Duchenne muscular dystrophy, X-linked centronuclear myopathy, and congenital muscular dystrophies.
- Translation and Collaboration: Involve small and medium-sized enterprises (SMEs) to facilitate the translation and exploitation of project outputs.
- Patient-Centric Approach: Collaborate closely with national and international patient advocacy groups, such as Muscular Dystrophy UK, Duchenne Data Foundation, and Parent Project aps, to ensure that the research remains patient-centered.
Approach
The MAGIC project employs a multidisciplinary approach that combines expertise in genetics, bioengineering, and advanced preclinical translation. It leverages state-of-the-art technologies, including microfabrication, microfluidics, and human stem cell differentiation, to create precise disease models and therapeutic vectors. These vectors are designed to express therapeutic genes specifically in muscle cells, reducing the risk of immune responses.
Furthermore, the project actively engages SMEs to facilitate the translation of research findings into practical applications. It also maintains a strong partnership with patient advocacy groups to incorporate the perspectives and needs of individuals affected by muscular dystrophies into the research process.
Through collaboration, innovation, and a focus on patient well-being, MAGIC aims to accelerate the development of effective treatments for muscular dystrophies, ultimately making a meaningful impact on the lives of those affected by these conditions.