Archive for November 2011

Harbor UCLA Neuro-Radiology Case 7

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Sterotactic and Functional Neurosurgery

Faculty: Peter Warnke; Ben Roitberg

Residents: Ricky Wong; Javed Khader-Eliyas; Ashley Ralston; Melissa Stamates; Mahua Dey

Articles:

1) LeWitt P, Rezai AR, Leehey, MA, Ojemann SG, Flaherty AW, Eskandar EN, Kostyk SK, Thomas K, Sarkar, A, Siddiqui MS, Tatter SB, Schwalb JM, Poston KL, Henderson JM, Kurlan RM, Richard IH, Van Meter L, Sapan CV, During M, Kaplitt MG, Feigin A. AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomized trial Lancet Neurology 2011; 10:309-19

Stamates: LeWitt et al. completed the first multi-center, double-blind randomized trial in gene therapy insertion for advanced Parkinson’s disease to demonstrate any therapeutic effect. Gene transfer of glutamic acid decarboxylase and subsequent GABA production in the subthalamic nucleus (STN) was proposed by the authors as superior treatment to DBS and subthalamotomy when medication fails to control PD symptoms. The authors performed a gene therapy trial under rigorous experimental standards, whereas most such studies are open-label. The relative standardization of stereotactic frame placement and microelectrode recording allowed each center to uniformly infuse the treatment vector (Adeno-Associated Virus – AAV) into each subject’s bilateral subthalamic nuclei. Controls only received partial thickness burr holes and the injection was simulated but did not take place.

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Harbor UCLA Neuro-Radiology Case 6

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