Adenoviral vector-mediated gene therapy for gliomas: coming of age.
Adenoviral vector-mediated gene therapy for gliomas: coming of age.
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DOI：
10.1517/14712598.2014.915307
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发表时间：
2014-09
影响因子：
4.6
通讯作者：
Lowenstein PR
中科院分区：
文献类型：
--
作者：
Castro MG;Candolfi M;Wilson TJ;Calinescu A;Paran C;Kamran N;Koschmann C;Moreno-Ayala MA;Assi H;Lowenstein PR
多形性胶质母细胞瘤 (GBM) 是成人中最常见的原发性脑肿瘤；它带来了悲观的预测。腺病毒载体 (Ad) 介导的基因转移正在被开发为 GBM 的一种有前景的治疗策略。临床前研究已经证明，将腺病毒注射到啮齿类动物的大脑和肿瘤块以及非人类灵长类动物的大脑中是安全有效的。重要的是，广告已在人体肿瘤切除腔内安全施用。 GBM 和 Ad 载体的背景；我们描述了 GBM 的基因治疗策略并讨论了组合方法的价值。最后我们讨论了使用腺病毒载体进行 GBM 的人体临床试验的结果。 Ad 载体的转导特性及其安全性，加上其实现高水平转基因表达的能力，使其成为治疗 GBM 的强大载体。最近基因治疗在视网膜疾病和全身脑代谢疾病治疗方面取得的成功，鼓励了恶性胶质瘤基因治疗的发展。令人兴奋的临床试验目前正在招募患者；尽管大型随机 III 期对照临床试验将为基因疗法治疗 GBM 的成功提供最终决定。
Glioblastoma multiforme (GBM) is the most common primary brain tumor in adults; it carries a dismal prognosis. Adenoviral vector (Ad)-mediated gene transfer is being developed as a promising therapeutic strategy for GBM. Preclinical studies have demonstrated safety and efficacy of adenovirus administration into the brain and tumor mass in rodents and into the non-human primates’ brain. Importantly Ads have been safely administered within the tumor resection cavity in humans. Background on GBM and Ad vectors; we describe gene therapy strategies for GBM and discuss the value of combination approaches. Finally we discuss the results of the human clinical trials for GBM that have used adenoviral vectors. The transduction characteristics of Ad vectors, and their safety profile, added to their capacity to achieve high levels of transgene expression have made them powerful vectors for the treatment of GBM. Recent gene therapy successes in the treatment of retinal diseases and systemic brain metabolic diseases, encourages the development of gene therapy for malignant glioma. Exciting clinical trials are currently recruiting patients; although it is large randomized phase III controlled clinical trials that will provide the final decision on the success of gene therapy for the treatment of GBM.
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影响因子：
4.8
作者：
Candolfi, Marianela;King, Gwendalyn D.;Castro, Maria G.
通讯作者：
Castro, Maria G.
影响因子：
5.7
作者：
Debinski, W;Gibo, DM
通讯作者：
Gibo, DM
影响因子：
4.8
作者：
DEBINSKI, W;OBIRI, NI;PURI, RK
通讯作者：
PURI, RK
DOI：
10.1158/1078-0432.ccr-13-2140
发表时间：
2014-03-15
期刊：
Clinical cancer research : an official journal of the American Association for Cancer Research
影响因子：
--
作者：
Candolfi M;Yagiz K;Wibowo M;Ahlzadeh GE;Puntel M;Ghiasi H;Kamran N;Paran C;Lowenstein PR;Castro MG
通讯作者：
Castro MG
影响因子：
45.3
作者：
Chiocca, E. Antonio;Aguilar, Laura K.;New, Pamela Z.
通讯作者：
New, Pamela Z.