Movement Disorders

Therapeutic Intervention Using Gene Modifying Strategies - Progress and Setbacks

Date/Time: Tuesday, October 25, 2022 - 11:00 AM – 12:30 PM
Track: Traditional Special Interest Group
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Therapeutic intervention using antisense oligonucleotide therapies in movement disorders and neurodegenerative diseases more broadly is an ever-increasing approach being taken in these diseases. The antisense oligonucleotide approach uses antisense drugs of various designs. The different designs allow ASOs to modulate RNA function either by targeting pre-mRNA or mature RNA for degradation. In addition, ASOs can modulate RNA function by altering splicing to increase specific RNA levels. Using an ASO approach in some of these diseases has been shown to be an effective therapeutic approach while some setbacks associated with the use of an ASO approach has also been observed. This SIG seeks to educate neurologists about the most recent findings using this approach. 

Chair: Michelle Gray, PhD

Co-Chair: Erin F. Stimming, MD, FAAN 


  • More knowledgeable about the antisense oligonucleotide technology being employed to treat various neurological diseases
  • Be better equipped to educate their patients concerning the types of clinical trials being performed using the antisense oligonucleotide technology
  • The feasibility of employing the technology in their clinical practice
  • Understand the risks associated with the use of antisense oligonucleotide technology

Treatments for Spinal Muscular Atrophy


Antisense Oligonucleotides for Spinocerbellar Ataxias


Tominersen in Huntington’s Disease


Reverse Translating Dystonia Assessment Following Neonatal Brain Injury Across Species: From Men to Mice

Poster Presenter: Kat Gemperli, Washington University of St. Louis

Distribution of α-Synuclein Oligomers in Brains of Parkinson’s Disease Patients with LRRK2 Mutations

Poster Presenter: Hiroaki Sekiya, MD, PhD, Mayo Clinic

Allele-Selective Inhibition of Mutant HTT Transcription Throughout the Brain After Subcutaneous Administration of a PATrOL™-Enabled Genome-Targeting Compound in the R6/2 Huntington’s Disease Transgenic

Poster Presenter: Robert Friedlander, MD, University of Pittsburgh