Mitochondrial disorders encompass a variety of uncommon, genetically diverse conditions resulting from mutations in the nuclear and mitochondrial genomes, which lead to dysfunctions of the mitochondria. Since most of these disorders are monogenic and due to mutations within the nuclear genes, gene replacement can be one of the most effective approaches. Protheragen offers its clients a complete platform for the development of gene therapy which facilitates research growth for the therapy of mitochondrial disorders.
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 provides a direct solution for the therapy of monogenic recessive diseases. Of considerable efficacy is the reintroduction gene strategy which entails the reintroduction of the wild-type form of a mutant gene or other therapeutic genes alongside a suitable delivery mechanism. The approach has shown favorable results in the management of highly debilitating genetic disorders like mitochondrial disease. Protheragen's high emphasis on preclinical research as a provider of life science services, we have developed an optimized research platform for gene therapies that focus on mitochondrial disease. Our scientists are experts in planning and executing a broad scope of validation assay services designed for your specific needs.
Gene Therapy Research and Design
Having some of the best scientists and researchers in gene therapy who have years of experience in the industry, we can perform deep research to understand the reasons behind mitochondrial diseases along with designing therapeutics for specific genetic mutations. We proficiently and precisely deliver gene editing and genomic alteration for repairing or replacing the mutated genes.
In Vitro and In Vivo Gene Delivery Studies
Our team can conduct in vivo and in vitro gene delivery studies using various therapeutic vectors in cell cultures as well as in animals for the safety and efficacy of gene therapy. We can test and demonstrate various therapeutic strategies using in vitro cell culture models. Also, we are fully capable of designing and conducting experiments with animals for gene therapy studies in various biological systems.
Carrier Optimization and Production
The selection of the proper vector is the most important element in the success of gene therapy. Our group focuses on developing and improving preexisting gene vectors to specific genes and therapies which enhances their delivery and stability. In addition, we also provide comprehensive large-scale production of carriers needed for preclinical trials.
Safety and Efficacy Evaluation
The safety and efficacy of gene therapy must be guaranteed in advance. Our group can make overall assessments of safety and efficacy including in vivo toxicity and immunogenicity evaluation. To guarantee the best range of safety and effectiveness for the provided therapy protocols, we make selected gene therapy protocols to meet high standards and carry out stringent experimental design and analysis.
Our Advantages
Professional Team
Advanced Technology
Comprehensive Services
Reliable Data and Results
Protheragen's team will work with you to offer curated solutions for the progression of gene therapy with respect to mitochondrial diseases. In case you are keen on our services concerning the development and therapy of mitochondrial diseases using gene therapy, feel free to contact us.
