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Abstract
With the emergence of high-throughput discovery platforms, robust preclinical small-animal models, and efficient clinical trial pipelines, it is becoming possible to envision a time when the treatment of human neurologic diseases will become personalized. The emergence of precision medicine will require the identification of subgroups of patients most likely to respond to specific biologically based therapies. This stratification only becomes possible when the determinants that contribute to disease heterogeneity become more fully elucidated. This review discusses the defining factors that underlie disease heterogeneity relevant to the potential for individualized brain tumor (optic pathway glioma) treatments arising in the common single-gene cancer predisposition syndrome, neurofibromatosis type 1 (NF1). In this regard, NF1 is posited as a model genetic condition to establish a workable paradigm for actualizing precision therapeutics for other neurologic disorders.
GLOSSARY
- cAMP=
- cyclic adenosine monophosphate;
- GEM=
- genetically engineered mouse;
- GWAS=
- genome-wide association studies;
- mTOR=
- mammalian target of rapamycin;
- NF1=
- neurofibromatosis type 1;
- NSC=
- neural stem cell;
- OPG=
- optic pathway glioma;
- PA=
- pilocytic astrocytoma;
- RGC=
- retinal ganglion cell
Footnotes
Go to Neurology.org for full disclosures. Funding information and disclosures deemed relevant by the author, if any, are provided at the end of the article.
- Received January 28, 2014.
- Accepted in final form April 9, 2014.
- © 2014 American Academy of Neurology
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