- Gene Therapies
Dystrogen is developing a new interfering RNA platform for treating patients with neurodegenerative genetic disorders.
Our platform applies a viral-based vector (created by unique nucleic acid molecule and expression vector), which has the ability to regulate the expression of an interfering RNA, responsible for long-term silencing effect of the mutated gene.
RNA interference (RNAi) is a natural cellular process taking part in the regulation of gene expression and it is also a mechanism of defense against viruses and mobile genomic elements. The basis for RNA interference is the participation of short double-stranded RNA molecules (dsRNA), 20-30 nucleotide long, in selective silencing of gene expression. These molecules together with specific proteins form RNA-Induced Silencing Complex (RISC) in cells. Active RISC recognizes and binds to complementary sequences within mRNA, and leads to degradation of the transcript or to translation inhibition, depending on the level of complementarity.
Application of Dystrogen Therapeutics proprietary vectors and promoters gives the possibility to regulate expression of interfering RNA of shRNA- and sh-miR-type, their selective delivery to the tissues of interest and most importantly a long-term silencing effect.
Candidates developed under RNAi Platform: