A single gene-silencing treatment can reverse symptoms associated with Huntington disease for a sustained period in both mouse and primate animal models of this neurodegenerative disorder (Kordasiewicz HB et al. Neuron. 2012;74:1031-1044).
The therapy, administered in a single infusion into the cerebral spinal fluid by scientists at the University of California at San Diego, consisted of single strands of DNA called antisense oligonucleotides. These selectively bind to and degrade the gene encoding huntingtin protein. When mutated, the gene causes toxic protein fragments to build up in the brain.
Hampton T. Targeting Huntington Disease. JAMA. 2012;308(5):444. doi:10.1001/jama.2012.9444