Researchers use our
technologies in their quest to
cure diseases like cancer,
HIV, and Parkinson’s

Gene-Editing Therapies for Parkinson’s Disease

Description

Parkinson’s disease is associated with the accumulation of alpha-synuclein aggregates in dopaminergic neurons of the substantia nigra pars compacta, leading to cell death, and resulting loss of motor control.

Our scientists developed a method for treating Parkinson’s disease by editing the gene that encodes alpha-synuclein to prevent aggregation of the encoded protein in affected cells.

Gene-Editing Therapies for Parkinson’s Disease is protected by two pending U.S. patents (with additional patents pending in other countries).


Example Applications

  • Employ multiple therapeutic strategies – monoallelic or biallelic inactivation of alpha-synuclein in vulnerable cells, altering alpha-synuclein mRNA splicing to generate non-aggregating forms by ablating splice acceptor/donor sites, etc.
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to brain in vivo