Mitochondrial diseases are a group of rare and heterogeneous disorders caused by genetic mutations in both the nuclear and mitochondrial genomes, resulting in impairments in mitochondrial function. Given that the majority of these conditions are monogenic and arise from mutations in nuclear genes, gene replacement emerges as an exceptionally promising therapeutic strategy. Protheragen provides clients with a comprehensive gene therapy development platform to accelerate the research process for targeting mitochondrial diseases.
Gene Therapies in Mitochondrial Disease
As research progresses, gene therapy holds great potential for addressing the underlying genetic defects in various mitochondrial disorders.
| Method |
Disease |
| AAV-mediated gene therapy |
Ant1−/− mouse model; Harlequin mouse; mouse model of Ethylmalonic encephalopathy (EE); mouse model of Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE); mpv17 knockout mouse model |
| CRISPR/Cas9 |
iPS cell model of CoQ10 deficiency (c.483G>C in COQ4 gene) |
Our Services
Gene therapy offers a compelling and straightforward therapeutic approach for monogenic recessive diseases. The reintroduction of the wild-type form of a mutant gene or other therapeutic genes, in conjunction with an appropriate delivery strategy, has demonstrated promising potential in the treatment of severe genetic disorders such as mitochondrial disease. As a leading provider of life science services, Protheragen applies a robust research platform for gene therapies specifically targeting mitochondrial diseases. Our team of scientists is proficient in designing and conducting a comprehensive range of validation assay services tailored to your specific objectives.
Gene Therapy Research and Design
Our team comprises professional gene therapy scientists and researchers with extensive expertise in the field. We possess the capability to conduct comprehensive investigations into the underlying causes of mitochondrial diseases, while also devising treatment strategies that specifically target genetic mutations. Leveraging advanced gene editing and delivery technologies, we strive to efficiently and accurately repair or replace mutated genes.
In Vitro and In Vivo Gene Delivery Studies
Our team is capable of conducting in vitro and in vivo gene delivery studies to assess the efficacy and safety of therapeutic vectors. Through the utilization of in vitro cell culture models, we can simulate and evaluate the impact of various therapeutic strategies. Furthermore, we are proficient in designing and implementing experiments using small animal models to assess the effects of gene therapy acro ss different biological systems.
Carrier Optimization and Production
Selecting the appropriate vector is crucial for the success of gene therapy. Our team specializes in enhancing and refining existing gene vectors tailored to specific genes and therapeutic targets, resulting in improved delivery efficiency and stability. Furthermore, we offer comprehensive large-scale carrier production services to meet the demands of preclinical trials.
Safety and Efficacy Evaluation
The safety and efficacy of gene therapy are of paramount importance. Our team is capable of conducting comprehensive assessments on safety and efficacy, including in vivo toxicity and immunogenicity studies. We employ rigorous experimental design and analytical methods to ensure that the offered gene therapy protocols fall within the range of optimal safety and efficacy.
Our Advantages
Professional Team
Advanced Technology
Comprehensive Services
Reliable Data and Results
The team of experts at Protheragen will collaborate with you to provide tailored solutions for advancing the development and application of gene therapy in mitochondrial diseases. If you are interested in our services related to gene therapy development and treatment for mitochondrial diseases, please do not hesitate to contact us.
