Gene therapy blockade of dorsal striatal p11 improves motor function and dyskinesia in parkinsonian mice

Abstract

Medications for Parkinson’s disease (PD) are designed to replace lost dopamine. Although effective, they often cause abnormal involuntary movements (AIMs), also called dyskinesias, which can be difficult to resolve without worsening PD symptoms. We report that p11, a small protein necessary for neurotransmitter receptor function, is critical to dopamine responses in a mouse PD model. Blocking p11 production in the dorsal striatum, a brain region that responds to dopamine and regulates movement, can improve forced movements and normalize spontaneous movements in parkinsonian mice while dramatically reducing AIMs after dopamine replacement therapy. Our data identify a new target for therapeutic development to both improve symptoms and reduce drug-related side effects in human PD.

Document Details

Document Type
Pub Defense Publication
Publication Date
Jan 19, 2016
Source ID
10.1073/pnas.1524387113

Entities

People

  • M. Angela Cenci
  • Margarita Arango-lievano
  • Michael G. Kaplitt
  • Paul Greengard
  • Per Svenningsson
  • Peter Morgenstern
  • Roberta Marongiu
  • Veronica Francardo
  • Xiaoqun Zhang

Organizations

  • Fisher Center for Alzheimer's Research Foundation
  • Freedom Together Foundation
  • Lund University
  • Swedish Research Council
  • The Rockefeller University
  • United States Department of Defense
  • Weill Cornell Medicine

Tags

Fields of Study

  • Biology
  • Psychology

Readers

  • Molecular and Cellular Biology
  • Neurodegenerative Parkinson's Disease and Rickettsial Disease handbook, including the data level of dopamine, BC, neurons, and PD.
  • Oncology

Technology Areas

  • Biotechnology
  • Biotechnology - Cancer Biotech