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Ractigen Reveals Preclinical Results of RNA Therapy for Duchenne Muscular Dystrophy

publication date: Nov 3, 2023

Ractigen Therapeutics, a Jiangsu RNA activation (RNAa) company, presented preclinical results of its short duplex RNA (saRNA) technology to develop a gene therapy for Duchenne muscular dystrophy (DMD). The saRNA targets activation of utrophin to increase transcription of endogenous genes, leading to restoration of their protein function. The company says saRNA offers the possibility of modulating traditionally undruggable targets, one of the few technologies that can treat diseases by stimulating the expression of therapeutic genes with insufficient expression. More details....

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