What Can RNA-Based Therapy Do for Monogenic Diseases?
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DOI: 10.3390/pharmaceutics15010260
Abstract: The use of RNA-based approaches to treat monogenic diseases (i.e., hereditary disorders caused by mutations in single genes) has been developed on different fronts. One approach uses small antisense oligonucleotides (ASOs) to modulate RNA processing… read more here.
Keywords: based therapy; rna based; therapy; monogenic diseases ... See more keywords