Gene therapy for Parkinson's disease.

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Gene therapy for Parkinson's disease. / Björklund, Tomas; Kordower, Jeffrey H.

I: Movement Disorders, Vol. 25 Suppl 1, 2010, s. S161-S173.

Forskningsoutput: TidskriftsbidragArtikel i vetenskaplig tidskrift

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Björklund, Tomas ; Kordower, Jeffrey H. / Gene therapy for Parkinson's disease. I: Movement Disorders. 2010 ; Vol. 25 Suppl 1. s. S161-S173.

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TY - JOUR

T1 - Gene therapy for Parkinson's disease.

AU - Björklund, Tomas

AU - Kordower, Jeffrey H

PY - 2010

Y1 - 2010

N2 - The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD-related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review.

AB - The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD-related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review.

U2 - 10.1002/mds.22785

DO - 10.1002/mds.22785

M3 - Article

C2 - 20187249

VL - 25 Suppl 1

SP - S161-S173

JO - Movement Disorders

JF - Movement Disorders

SN - 0885-3185

ER -