Dynamin-2 pre-messenger RNA (DNM2 pre-mRNA)

DNM2 pre-mRNA is the newly transcribed RNA intermediate of the *DNM2* gene that is subject to alternative splicing, generating multiple isoforms of dynamin-2, a large mechanochemical GTPase. The splicing process can be targeted therapeutically to correct gain-of-function mutations responsible for autosomal dominant centronuclear myopathy and Charcot–Marie–Tooth disease. Such therapies include antisense oligonucleotides and trans-splicing approaches that modify the pre-mRNA before it is processed into mature mRNA[3][6]. The pre-mRNA itself is not a mature protein or classical therapeutic protein target, but is relevant to RNA-modulation therapies for genetic disease.

Other names

DNM2 precursor mRNADynamin-2 transcriptDNM2 gene transcript

Antisense oligonucleotides lead to mRNA degradation or alteration of splicing, reducing dynamin-2 protein levels. Spliceosome-mediated RNA trans-splicing corrects pathogenic mutations at the pre-mRNA level.