Muscle-targeting Adenovirus Vector Construction Service
Creative Biolabs' research team and technical staff have established a perfect adenovirus vector (AV) construction platform through unremitting efforts. Our AV construction technology is comprehensive and advanced to meet various experimental needs. We have added a targeting sequence to the AV to enable efficient delivery of genetic material to target organs. Moreover, we have designed muscle-targeted AV specifically for muscle-related genetic diseases, which can specifically transfer genes of your interest to muscle tissue to help your gene therapy research. No matter what difficulties you have encountered in the relevant research work, you can contact us to get your customized service.
Introduction of Muscle Disease and Gene Therapy
Hereditary muscle diseases are generally caused by dysfunction of skeletal muscle and heart dysfunction, with high morbidity and mortality, and most related diseases lack effective treatment, indicating that there are still many existing medical technologies need to improve. Gene therapy offers a promising therapeutic perspective for many patients with genetic diseases, including muscle disease. Muscle-targeted gene therapy is also the first approved gene therapy program.
Most conventional hereditary muscle diseases are caused by defects in single genes, such as Duchenne muscular dystrophy (DMD) caused by mutations in the dystrophin (DYS) gene. Nowadays, gene therapy is already a very common method in the treatment of this disease. The usual practice is to transfer the truncated DYS gene into muscle cells using an adeno-associated viral vectors (AAVs). In addition, considering the secretory function of muscle, muscle is also a very attractive target for gene transfer in the treatment of other diseases. However, despite the bright prospects of gene therapy for muscle, how to efficiently express specific therapeutic genes in skeletal muscle has been a challenging task. Although the current clinical treatment of DMD proves that it is feasible to repair hereditary diseases through gene therapy, the overall therapeutic effect is still limited. Increasing the dose of the recombinant vectors can increase the efficacy, but it also increases the risk of the immune response. To overcome these limitations, more efficient gene therapy vectors must be developed to achieve high and broad muscle-specific expression at a safe carrier dose.
Muscle-targeting Adenovirus Vector
Adenovirus is one of the most commonly used gene transfer vectors. Due to its wide existence and adaptability, adenovirus is regarded as one of the most potential recombinant vectors for gene therapy in humans. Because adenovirus is a type of non-enveloped double-stranded DNA virus, it has an advantage in the selectivity of transfection and the lower threat to human health. More than 400 gene therapy experiments have been or are currently using human adenoviral vectors. Most of these experiments are directed at some hereditary diseases or tumors, and some vectors carry antigenic protein genes for use as vaccines. In gene therapy, some normal genes are introduced into a patient by the adenoviral vector to express non-mutant proteins to correct gene defects.
In the field of treatment of muscle diseases, the basal transfer efficiency of adenovirus was not ideal due to the lack of suitable virus-attached receptors on the surface of muscle fibers. The commonly used serotype 5 adenoviral vector could not efficiently transduce genes into adult muscle tissue. However, recent studies have found that modification of the viral capsid can significantly improve the gene delivery capacity of adenovirus to skeletal muscle. First-generation and helper-dependent serotype 5 adenoviral vectors featuring the serotype 3 knob (5/3) can not only effectively transfer full-length dystrophin proteins to the muscles of MDX mice, but also show stable gene expression for up to 1 year.
Creative Biolabs' scientists have worked hard to overcome the shortcomings after years of research and modification of adenoviral vectors. The adenoviral vectors we provide have the world's leading applicability and efficiency. If you have any question or have any difficulty, you can contact us by email or send us an inquiry to find a complete solution.
