Protein Replacement for Rett Syndrome
While the Gene Therapy Consortium is working to overcome the challenges of gene therapy for Rett (for example, the overexpression issue) RSRT is also supporting synergistic strategies in parallel.
One such strategy is protein replacement. Rather than delivering genes, this approach delivers the protein that the gene encodes. In order to deliver the MeCP2 protein to the brain we must penetrate the blood brain barrier (BBB), the protective dynamic interface that separates the brain from the circulatory system and protects the central nervous system from potentially harmful chemicals while regulating transport of essential molecules and maintaining a stable environment.
ArmaGen’s platform technology takes advantage of the body’s natural system to non-invasively deliver drugs across the BBB. The BBB selectively allows vital nutrients to pass from the bloodstream to the brain, through the presence of receptors that enable the entry of compounds such as insulin, transferrin (protein that transports iron) and low-density lipoproteins (LRP1, proteins that transport fat). ArmaGen’s approach targets the same receptors that transport these compounds to the brain.
Armagen’s scientists will fuse molecules to the MeCP2 protein that will allow it to be recognized and pumped across the blood brain barrier. The advantage to this approach is that protein can be titrated to a much greater degree than gene therapy.