2 results match your criteria: "Mission Center Building Room 226[Affiliation]"

Gene therapy for Parkinson's disease: from non-human primates to humans.

Curr Opin Mol Ther

October 2010

University of California San Francisco, Neurosurgery Department, 1855 Folsom Street, Mission Center Building Room 226, San Francisco, CA 94103, USA.

Gene therapy strategies in non-human primate models of Parkinson's disease (PD) are beginning to produce results consistently, and have been successfully translated to clinical trials. Although not all of the therapeutic efforts based on gene therapy have demonstrated clinical efficacy, the stereotactic techniques and at least three different beneficial genes that have been delivered to patients have been proven to be safe. The adeno-associated virus has been used as an effective and safe delivery vehicle for the first three, single therapeutic transgenes (ie, glutamic acid decarboxylase, aromatic l-amino acid decarboxylase, and neurturin) to be tested in trials.

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Article Synopsis
  • Treatment of malignant gliomas is challenging, with current methods yielding less than one year median survival; a new technique, convection-enhanced delivery (CED), using a nanoparticle liposome with topotecan shows promise.
  • CED of liposomal topotecan allows for prolonged retention in brain tissue (1.5 days) and effective accumulation around blood vessels, enhancing its antiangiogenic effects and topoisomerase I inhibition.
  • In rat models, CED with liposomal topotecan significantly improved survival rates and treatment efficacy compared to free topotecan, suggesting that this method could be effective for treating brain tumors.
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