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Therapeutic targets for neuroblastomas
Abstract: Introduction: Neuroblastoma (NB) is the most common and deadly solid tumor in children. Despite recent improvements, the long-term outlook for high-risk NB is still < 50%. Further, there is considerable short- and long-term toxicity. More effective, less toxic therapy is needed, and the development of targeted therapies offers great promise.

Areas covered: Relevant literature was reviewed to identify current and future therapeutic targets that are critical to malignant transformation and progression of NB. The potential or actual NB therapeutic targets are classified into four categories: i) genes activated by amplification, mutation, translocation or autocrine overexpression; ii) genes inactivated by deletion, mutation or epigenetic silencing; iii) membrane-associated genes expressed on most NBs but few other tissues; or iv) common target genes relevant to NB as well as other tumors.

Expert opinion: Therapeutic approaches have been developed to some of these targets, but many remain untargeted at the present time. It is unlikely that single targeted agents will be sufficient for long-term cure, at least for high-risk NBs. The challenge will be how to integrate targeted agents with each other and with conventional therapy to enhance their efficacy, while simultaneously reducing systemic toxicity.
Keywords:ARID1A/B  ATRX  AURKA  BCL2  CAMTA1  CASZ1  CHD5  CHEK1/CHK1  FOXR1  GD2  HDAC  KIF1Bβ  MCL1  MYCN  NCAM  NET  neuroblastoma  NTRK1/TrkA  NTRK2/TrkB  ODC  PTPN11  RAS  TP53/P53
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