Neurofibromatosis-related tumors: emerging biology and therapies.
Neurofibromatosis-related tumors: emerging biology and therapies.
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DOI：
10.1097/mop.0000000000000169
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发表时间：
2015-02
影响因子：
3.6
通讯作者：
Ferner RE
中科院分区：
文献类型：
--
作者：
Karajannis MA;Ferner RE
在过去的十年里，对肿瘤抑制因子神经纤维素(NF1)和Merlin(NF2)的生物学功能已经有了实质性的了解。本综述的目的是强调神经纤维瘤病1型(NF1)和神经纤维瘤病2型(NF2)生物学方面的一些主要进展，因为它们与这些疾病的新疗法的开发有关。近年来，越来越复杂的临床前模型的发展为合理开发NF1和NF2相关肿瘤的分子靶向治疗提供了平台，例如在国防部赞助的神经纤维瘤病临床试验联盟(NFCTC)内。分子靶向治疗的临床试验已经成为NF患者的现实，并有望改善受这些疾病影响的个人的发病率和死亡率。
Over the past decade, substantial insight into the biological function of the tumor suppressors neurofibromin (NF1) and Merlin (NF2) has been gained. The purpose of this review is to highlight some of the major advances in the biology of neurofibromatosis type 1 (NF1) and neurofibromatosis type 2 (NF2) as they relate to the development of novel therapies for these disorders. The development of increasingly sophisticated preclinical models over the recent years has provided the platform from which to rationally develop molecular targeted therapies for both NF1 and NF2 related tumors, such as within the Department of Defense-sponsored Neurofibromatosis Clinical Trials Consortium (NFCTC). Clinical trials with molecular targeted therapies have become a reality for NF patients, and hold substantial promise for improving the morbidity and mortality of individuals affected with these disorders.