Diffuse Intrinsic Pontine Glioma: New Pathophysiological Insights and Emerging Therapeutic Targets.
Diffuse Intrinsic Pontine Glioma: New Pathophysiological Insights and Emerging Therapeutic Targets.
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DOI：
10.2174/1570159x14666160509123229
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发表时间：
2017
影响因子：
5.3
通讯作者：
Monje M
中科院分区：
文献类型：
--
作者：
Johung TB;Monje M
弥漫性桥脑胶质瘤(DIPG)是儿童脑肿瘤相关死亡的主要原因，中位生存期不到一年。尽管进行了数十年的临床试验，但自30多年前引入放射治疗以来，预后并未得到改善。回顾DIPG的临床特征和当前的治疗挑战，并讨论推动DIPG发病的独特基因组和表观基因组机制的新见解，为确定治疗靶点提供新的机会。近年来，用于临床前研究的活检和快速尸检组织样本的可用性增加，加上新的基因组和表观基因组图谱工具的出现，使我们对DIPG疾病机制的理解有了显著的进步。此外，对DIPG发育背景的深入了解有助于揭示儿童大脑微环境中的治疗靶点。
Diffuse Intrinsic Pontine Glioma (DIPG) is the leading cause of brain tumor-related death in children, with median survival of less than one year. Despite decades of clinical trials, there has been no improvement in prognosis since the introduction of radiotherapy over thirty years ago. To review the clinical features and current treatment challenges of DIPG, and discuss emerging insights into the unique genomic and epigenomic mechanisms driving DIPG pathogenesis that present new opportunities for the identification of therapeutic targets. In recent years, an increased availability of biopsy and rapid autopsy tissue samples for preclinical investigation has combined with the advent of new genomic and epigenomic profiling tools to yield remarkable advancements in our understanding of DIPG disease mechanisms. As well, a deeper understanding of the developmental context of DIPG is shedding light on therapeutic targets in the microenvironment of the childhood brain.