Is gene therapy safe and effective in the treatment of Parkinson disease (PD)?

Updated: Jan 24, 2019
  • Author: Robert A Hauser, MD, MBA; Chief Editor: Selim R Benbadis, MD  more...
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Answer

Several studies have demonstrated the safety of gene therapy as a treatment for Parkinson disease, and larger studies have been initiated to examine the efficacy of this procedure. Three investigational strategies that use gene transfer for targeted protein expression are as follows [99] :

  • Improving dopamine availability to the striatum using more continuous delivery,

  • Reducing STN activity with local induction of gamma-aminobutryic acid (GABA) expression

  • Protection/restoration of nigrostriatal neuronal function with trophic factor expression

A double-blind, phase II, randomized, controlled trial of gene delivery of the trophic factor neurturin via an adeno-associated type-2 vector (AAV2) in Parkinson patients aged 30-75 years suggested mild efficacy. Further studies are ongoing. [100]


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