Two molecular control factors play a decisive role in what is known as splicing, the cutting and assembly of mature messenger RNA—a prerequisite for protein synthesis in the cell. The poorly ...
RNA splicing involves the removal of non-coding introns and the joining of coding exons in precursor mRNA (pre-mRNA) to produce mature messenger RNA (mRNA). Splicing changes can lead to diseases, ...
Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ...