Cell-Based Model Development Service

Mitochondrial disorders, often attributed to mutations in mitochondrial DNA (mtDNA), present significant challenges in comprehending their mechanisms and discovering targeted therapies. Protheragen aims to address these challenges by providing advanced cell models for the study of mitochondrial diseases, ultimately facilitating drug discovery and therapeutic advancements.

Overview of Mitochondrial Diseases

Mitochondrial diseases arise from dysfunctions in oxidative phosphorylation, impacting high-energy-demanding tissues such as the heart, brain, and skeletal muscles. mtDNA mutations, which encode crucial components of the electron transport chain complexes, significantly contribute to mitochondrial disorders. Despite advancements made thus far, establishing effective cellular models for studying these diseases remains a challenge that hinders progress in targeted drug discovery.

Fig.1 Modeling tools for mitochondrial diseases. (Tolle, I., et al., 2023)

Our Services

With our extensively tested technical expertise in gene editing and mitochondrial disease research, Protheragen offers a comprehensive service for the development of cell-based models to accurately model mitochondrial diseases based on client requirements. Our services encompass various aspects of cell-based model development, including but not limited to the following.

Immortalized Lymphoblastoid Cells

The immortalized lymphoblastoid cell, generated through Epstein Barr virus transformation, offers significant advantages in drug screening for mitochondrial diseases. These cells, derived from peripheral blood, effectively maintain their inherent biological properties and serve as a robust platform for large-scale drug discovery efforts.

Fibroblasts

Obtained from muscle biopsy-derived fibroblasts, these cells offer a valuable resource for drug discovery. Patient-specific fibroblasts facilitate the investigation of mitochondrial disorders, particularly those involving complex I deficiencies, providing insights into potential therapies and personalized medicine.

Cytoplasmic Hybrid (Cybrid) Cells

These cybrid cells, generated by fusing patient-derived cytoplasts with ρ0 cells lacking mtDNA, effectively eliminate interference caused by different nuclear backgrounds. Cybrid cells serve as invaluable models for investigating the impact of mtDNA mutations on mitochondrial function, thereby facilitating drug discovery efforts.

iPSCs and Differentiation Cells

Reprogrammed from somatic cells, induced pluripotent stem cells (iPSCs) offer a groundbreaking approach to modeling mitochondrial diseases. Our iPSC-derived cell lines, including cardiomyocytes and neural progenitor cells, faithfully recapitulate disease-specific pathology, providing an optimal platform for drug discovery and elucidating disease mechanisms.

Our Advantages

As technology advances, intelligent screening methods, and improved cell models are expected to further accelerate mitochondrial drug discovery, our cell-based model development service is dedicated to accelerating the comprehension and treatment of mitochondrial diseases. Through offering state-of-the-art cell models and fostering collaborative research, we strive to make significant contributions to the field of mitochondrial drug discovery. For any inquiries or potential collaborations, please do not hesitate to contact us.