AAV2-neurturin for Parkinson’s disease: What lessons have we learned?

Research output: Chapter in Book/Report/Conference proceedingChapter

14 Scopus citations

Abstract

The dream that trophic factors could be effectively delivered and potently forestall and reverse the symptoms of Parkinson’s disease (PD) has yet to be realized. Research in this area has been active for 20 years, but after much work, the prospects for utilizing trophic factors in the treatment of PD are currently dim. Millions of dollars have been spent, numerous academic, foundation, and government resources have been invested, and hundreds of patient research volunteers have contributed their time and hope to this effort without a therapeutic breakthrough. As a scientist who has journeyed these events from the beginning and participated in many of the decisions that navigated this field, I consider it important for the movement disorder scientific community to reflect on the evolution of thought and to participate in the dialog over whether the investments were worthwhile. The most studied group of trophic factor for PD is the glial cell derived family of ligands, of which glial cell derived neurotrophic factor (GDNF) and neurturin are members, and are the best studied. I trace the development of these factors chronologically with commentary on the key decision-making points. Before we collectively invest further, I offer this scientific reflection on the past and offer my own view on the next steps of research in the field of neurotrophins as potential therapeutic agents in PD.

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages485-490
Number of pages6
DOIs
StatePublished - 2016
Externally publishedYes

Publication series

NameMethods in Molecular Biology
Volume1382
ISSN (Print)1064-3745

Keywords

  • AAV
  • Clinical trial
  • GDNF
  • Neurturin
  • Nigrostriatal system
  • Parkinson’s disease

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics

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