Which of these diseases is likely one for which an RNA drug

Which of these diseases is likely one for which an RNA drug similar to the one we discussed that was just approved for SMA. Hutchinson-Gilford progeria syndrome, which is caused by a mutation in the LMNA gene that creates a new splice site donor. This new splice site leads to a truncated protein. Myotonic dystrophy, which is caused by an expansion of CIG repeats in the 3\'-untranslated region of DMPK. Transcripts containing an expanded CUG repeat are retained in the nucleus, which leads to reduced expression Of DMPK protein. Shwachman- Diamond syndrome, which caused by a recombination between the gene SBDS and a pseudogene of SBDS that is distally located. The gene SBDS has clearly known function. X-linked dyskeratosis congenita, which is causea by SNPs that lead to changes in highly-conserved amino acids in the DRC1 gene.

Solution

The answer is Myotonic dystrophy for which using IONIS-DMPKRx antisense technology that targets the molecule believed to be responsible for causing toxicity in people. The mRNA is known as ‘toxic RNA’ containing a large repeat made from the DMPK gene. This mRNA targets DMPK RNA strongly and penetrate different tissues more efficiently.

The treatment of DM also uses RNA drug similar to used in treatment of SMA.