Biopharmaceutical company Seelos Therapeutics in conjunction with researchers at Duke University School of Medicine announced promising results for an epigenome-editing therapy designed to reduce alpha-synuclein levels.
The experimental treatment, SLS-004, delivers CRISPR-dCas9 through a modified virus. Rather than editing genes by altering DNA, CRISPR-dCas9 allows for the regulation of a gene without changing it. In this case, the targeted gene is SCNA, which is responsible for the production of the protein alpha-synuclein. In excess, alpha-synuclein clumps together into Lewy bodies and causes Parkinson’s disease.
Researchers injected two forms of SLS-004 directly into the left hemispheres of the brains of healthy mice. One form carried the epigenome modifier DNMT3A and resulted in a 20% drop in alpha-synuclein levels relative to the right hemisphere. The other form carried the repressor KRAB-MeCp2 and resulted in a 40% drop in alpha-synuclein relative to the right hemisphere. Control mice showed no difference in alpha-synuclein levels in the left vs right hemispheres.
Duke researchers plan to continue preclinical studies of SLS-004 as part of a sponsored research agreement with Seelos with the hope that it can lead to targeted therapies for Parkinson’s disease.