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 ...
Trans-splicing is a unique process in gene expression where a short sequence, known as a spliced leader (SL), is added to the 5' end of mRNA molecules. This mechanism is particularly significant in ...
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