合作類(lèi)型：技術(shù)     行業(yè)類(lèi)別: 醫(yī)藥研發(fā)    過(guò)期時(shí)間：2016年10月09日   

荷蘭一所醫(yī)藥大學(xué)已研發(fā)出一種用于治療肌營(yíng)養(yǎng)不良和其他疾病的新方法，目前正在尋找對(duì)治療肌營(yíng)養(yǎng)不良癥感興趣的制藥和生物技術(shù)公司。

英文原文：A Dutch medical university has developed new method that uses exon skipping in ALK4 and ALK5 for treatment of muscular dystrophies and other disorders. They are interested in collaborations with pharmaceutical and biotech companies for researching and developing treatments for muscular dystrophy. TGF-beta and myostatin are cytokines that are potent muscle growth inhibitors and are involved in the regulation of muscle cell differentiation. These cytokines bind to Acvr1b (ALK4) or Tgfbr1 (ALK5) to signal and elicit their functions. This Dutch medical university has developed a new exon skipping strategy to interfere in these signalling processes. This involves developing antisense oligonucleotides (AONs) that disrupt encoding for the ligand binding or the kinase domains in ALK4 or ALK5. As a result, the target cells generate non-functional receptors, and abrogated TGF-beta/myostatin signalling. Muscular Dystrophy’s pathophysiology involves muscle damage, fibrosis and impaired muscle regeneration, whilst sarcopenia and cachexia are conditions characterized by a significant loss of skeletal muscle. The treatment of these diseases could significantly benefit from a method to inhibit the muscular atrophy and fibrosis, thereby preventing the recurrence of the disease. ? Specific inhibition of target receptor genes. ? Therapeutic potential in several disorders associated with up-regulated TGF-beta signalling. ? The use of AONs to generate non functional TGF-beta/myostatin receptors could potentially add a beneficial value to the dystrophin exon skipping to ameliorate dystrophic phenotype.