Cell-Based Model Development Service

Mitochondrial disorders, primarily caused by mutations in mitochondrial DNA (mtDNA), are challenging to understand and difficult to develop precise therapeutics. To overcome these hurdles, Protheragen supplies advanced cellular models for the research of mitochondrial diseases, which, in the end, should speed up drug and therapeutic development.

Overview of Mitochondrial Diseases

Mitochondrial disorders occur due to problems with oxidative phosphorylation for highly energetic demanding tissues, for example, those of the heart, brain, and skeletal muscles. Mitochondrial disorders are in part due to mutations in the mtDNA, which also hold significant value due to their relations with the components of the electron transport chain complexes. There have been certain advancements; however, the progress of targeted drug development suffers because of the inability to provide stringent cellular models for the exploration of these diseases.

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

Our Services

Utilizing our experience in gene editing and mitochondrial diseases research, Protheragen has developed an accurate, comprehensive service for developing cell-based models that meet client specifications. Our services span all elements of cell-based model development, including but not limited to the following.

Immortalized Lymphoblastoid Cells

The immortalized lymphoblastoid cells transformed by the Epstein-Barr virus are instrumental for drug screening against mitochondrial ailments. Not only do the cells maintain their biological functions, but they serve as a great platform for drug discovery and development, thus achieving the goal of drug screening on a larger scale.

Fibroblasts

These cells are derived from muscle biopsies taken from individuals suffering from specific mitochondrial disorders involving complex deficiencies. The fibroblasts are essential for drug discovery and facilitate deep research involving the potential therapies along with personal drug aimed toward the specific individual.

Cytoplasmic Hybrid (Cybrid) Cells

These hybrid cells are created by fusing cytoplasts from the patient-derived cytoplasts with ρ0 cells lacking mitochondrial DNA. This method allows us to overcome the challenge posed by variations in nuclear backgrounds. Cybrid cells are effective in studying the impact of mtDNA mutations on the functions of mitochondria and enhancing drug discovery.

iPSCs and Differentiation Cells

Induced pluripotent stem cells (iPSCs) as a result of reprogrammed from somatic cells are the groundbreaking approach to modeling mitochondrial diseases. Our iPSC-derived cell lines, such as cardiomyocytes and neural progenitor cells, are an ideal source for drug discovery and studying disease mechanisms.

Our Advantages

With the continuously evolving advancements in attained technology, increased screening methods, as well as improved cellular models, the acceleration of drug discovery within the mitochondria is within reach, which is why our model development service is focused on expediting the understanding of mitochondria-related disorders. By providing novel cell models and encouraging collaboration, we hope to meaningfully enhance the field of mitochondrial drug development. For any inquiries or potential collaborations, please do not hesitate to contact us.