About
Dystrogen Gene Therapies 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 produce an interfering RNA, responsible for long-term silencing of the mutated gene.
The therapeutic goal is to inhibit the production of the mutant protein, leaving the normal protein intact (allele-selectivity). Our molecule targets expanded CAG repeat tracts in transcripts and inhibits production of toxic proteins with polyQ domains.
RNAi Technology
RNA interference (RNAi) is a natural cellular process which takes 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 Gene Therapies 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.
Huntington's Diseases Therapy
Competitive advantages:
GENE THERAPIES for HD
CAG sh-miRNA
Technology
RNAi
Acting on the mutant gene
Targets 100% HD patients
Long-term silencing
Universal therapy for other diseases with CAG expansion
GENE THERAPIES for HD
Exon-targeting
Technology
RNAi
Acting on the mutant gene
Targets 100% HD patients
Long-term silencing
Universal therapy for other diseases with CAG expansion
GENE THERAPIES for HD
SNP- targeting
Technology
Antisense
Acting on the mutant gene
Targets 100% HD patients
Long-term silencing
Universal therapy for other diseases with CAG expansion
Dystrogen Gene Therapies Pipeline
Dystrogen Gene Therapies is developing an RNAi Platform focused on polyglutamine (polyQ) disorders caused by trinucleotide repeat expansion in associated genes. These include e.g. Huntington's disease, Dentatorubral Pallidoluysian Atrophy (DRPLA), Spinocerebellar Ataxia Type 3 (SCA3) - otherwise Machado-Joseph and Spinocerebellar Ataxia Type 7 (SCA7).
