Development of AAV2-hAADC Gene Therapy for Parkinson's Disease designed to Enhance Dopamine Metabolism

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概要

• 論文の詳細を見る

• Dopamine, the major neurotransmitter depleted in Parkinson's disease, can be synthesized and regulated in vivo with a combination of intra-striatal AAV2-hAADC gene therapy and administration of the dopamine precursor L-dopa. When tested in MPTP-lesioned monkeys, this approach resulted in long-term improvement in clinical rating scores, significantly lowered L-dopa requirements, and a reduction of L-dopa-induced side effects. Positron emission tomography with [^<18>F]-FMT confirmed persistent AADC activity, demonstrating for the first time that infusion of AAV2 vector into primate brain results in at least 6 years of transgene expression. Armed with these data, a Phase 1 open-label does-escalation trial is now underway and interim results are expected in the near future.

• 2008-04-20

著者

• Forsayeth John

  Department Of Neurological Surgery University Of California San Francisco

• Forsayeth John

  Departmemt Of Neurosurgery University Of California

• Bankiewicz KS

  Department of Neurological Surgery, University of California San Francisco

関連論文

• 3. AAV/AADC Gene Therapy for Parkinson's Disease(LS1-4 Surgical Management of Involuntary Movement Disorders, The 27^ Annual Meeting of The Japanese Congress of Neurological Surgeons)
• Development of AAV2-hAADC Gene Therapy for Parkinson's Disease designed to Enhance Dopamine Metabolism

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