Gene Therapy for the Treatment of Neurological Disorders: Central Nervous System Neoplasms

基因治疗治疗神经系统疾病:中枢神经系统肿瘤

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作者:Neha Kamran, Marianela Candolfi, Gregory J Baker, Mariela Moreno Ayala, Marta Dzaman, Pedro R Lowenstein, Maria G Castro

Abstract

Glioblastoma multiforme (GBM) is the most common primary brain tumor in adults with a median survival of 16.2-21.2 months post diagnosis (Stupp et al., N Engl J Med 352(10): 987-996, 2005). Because of its location, complete surgical resection is impossible; additionally because GBM is also resistant to chemotherapeutic and radiotherapy approaches, development of novel therapies is urgently needed. In this chapter we describe the development of preclinical animal models and a conditionally cytotoxic and immune-stimulatory gene therapy strategy that successfully causes tumor regression in several rodent GBM models.

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