New potential treatment of tumor cachexia is coming

Recently, the international academic journal Molecular Therapy published the latest study results of Ding Qiurong Research Group from Chinese Academy of Sciences, Shanghai Institute of Life Sciences Institute of Nutrition Science, and the paper is titled "Prevention of Muscle Wasting by CRISPR / Cas9- mediated Disruption of Myostatin In Vivo". The aim of this study through recombinant dog proteins is to treat myosinophilia by CRISPR in muscle-specific target-knockout myostatin in response to severe muscular dystrophy in cancer patients.

Cachexia can be found in a variety of diseases, of which the cachexia associated with the tumor is most common. Tumor cachexia is a consumptive syndrome shown in many cancer patients, especially in late stages of disease. Although there are sufficient nutrient intake, the substantial loss of skeletal muscle also exist (with or without adiposity), leading to a serious decline in quality of life and survival.

In this study, under the guidance of researcher Ding Qiurong, Wei Yuda, a graduate student achieved used SaCRISPR / Cas9 to target the myostatin signaling pathways that are activated the effect of partly alleviating the cachexia in cachexia through the myostatin in body-targeted knockout muscles aiming at inducing myostatin signaling pathway. The main reasons for choosing myostatin as the target are: 1) myostatin signal pathway is the negative regulator of muscle growth and development. In addition to the muscular system, no serious adverse effects have been found in the natural individuals with myostatin function deletion mutations, and the safety of targeting has been guaranteed. 2) Early serial mice and clinical trials have demonstrated that the myostatin signaling pathway in cachexia (3) myostatin is mainly secreted by the muscle cells. The main mode of action is autocrine / paracrine mode, so the reduction in myostatin concentration in the muscle microenvironment can preserve some of the muscle function in the presence of cachexia.

Based on this, Wei et al. made use of muscle-specific promoter to start the expression of SaCas9 (staphylococcus aureus cas9) and then injected into the glandular-associated virus vector (AAV). After packed in mouse myostatin protein, significant recovery of skeletal muscle function was observed in the tumor-induced cachexia mice model (9% increase in mean skeletal muscle weight, 25% increase in average grip strength, and a slice at the injection site, compared to non-targeted mice staining found muscle fiber atrophy was significantly alleviated). This study as a confirmatory experiment suggests that the use of gene editing specific target knockout myostatin in muscle tissue can be used as a potential gene therapy for cachexia control and can further improve the in-vivo targeting efficiency, and a comprehensive assessment of targeted safety will contribute to its true clinical transformation. Flarebio offers recombinant proteins of great quality including recombinant CDH2 at good prices.