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Neurofibromatosis type 1 and optic pathway glioma: Molecular interplay and therapeutic insights
Author(s) -
Khatua Soumen,
Gutmann David H.,
Packer Roger J.
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26838
Subject(s) - neurofibromatosis , medicine , stromal cell , neurofibromin 1 , glioma , cancer research , neurofibromatosis type 2 , central nervous system , genetically engineered , pathology , biology , genetics , gene
Children with neurofibromatosis type 1 (NF1) are predisposed to develop central nervous system neoplasms, the most common of which are low‐grade gliomas (LGGs). The absence of human NF1 associated LGG‐derived cell lines, coupled with an inability to generate patient‐derived xenograft models, represents barriers to profile molecularly targeted therapies for these tumors. Thus, genetically engineered mouse models have been identified to evaluate the interplay between Nf1 ‐deficient tumor cells and nonneoplastic stromal cells to evaluate potential therapies for these neoplasms. Future treatments might also consider targeting the nonneoplastic cells in NF1–LGGs to reduce tumor growth and neurologic morbidity in affected children.