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Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder that results from germline mutations of the NF1 gene, creating a predisposition to low-grade gliomas (LGGs; pilocytic astrocytoma) in young children. Insufficient data and resources represent major challenges to identifying the best po...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5167692/ https://www.ncbi.nlm.nih.gov/pubmed/28066715 http://dx.doi.org/10.3389/fonc.2016.00259 |
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author | Ricker, Cora A. Pan, Yuan Gutmann, David H. Keller, Charles |
author_facet | Ricker, Cora A. Pan, Yuan Gutmann, David H. Keller, Charles |
author_sort | Ricker, Cora A. |
collection | PubMed |
description | Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder that results from germline mutations of the NF1 gene, creating a predisposition to low-grade gliomas (LGGs; pilocytic astrocytoma) in young children. Insufficient data and resources represent major challenges to identifying the best possible drug therapies for children with this tumor. Herein, we summarize the currently available cell lines, genetically engineered mouse models, and therapeutic targets for these LGGs. Conspicuously absent are human tumor-derived cell lines or patient-derived xenograft models for NF1-LGG. New collaborative initiatives between patients and their families, research groups, and pharmaceutical companies are needed to create transformative resources and broaden the knowledge base relevant to identifying cooperating genetic drivers and possible drug therapeutics for this common pediatric brain tumor. |
format | Online Article Text |
id | pubmed-5167692 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-51676922017-01-06 Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma Ricker, Cora A. Pan, Yuan Gutmann, David H. Keller, Charles Front Oncol Oncology Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder that results from germline mutations of the NF1 gene, creating a predisposition to low-grade gliomas (LGGs; pilocytic astrocytoma) in young children. Insufficient data and resources represent major challenges to identifying the best possible drug therapies for children with this tumor. Herein, we summarize the currently available cell lines, genetically engineered mouse models, and therapeutic targets for these LGGs. Conspicuously absent are human tumor-derived cell lines or patient-derived xenograft models for NF1-LGG. New collaborative initiatives between patients and their families, research groups, and pharmaceutical companies are needed to create transformative resources and broaden the knowledge base relevant to identifying cooperating genetic drivers and possible drug therapeutics for this common pediatric brain tumor. Frontiers Media S.A. 2016-12-20 /pmc/articles/PMC5167692/ /pubmed/28066715 http://dx.doi.org/10.3389/fonc.2016.00259 Text en Copyright © 2016 Ricker, Pan, Gutmann and Keller. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Oncology Ricker, Cora A. Pan, Yuan Gutmann, David H. Keller, Charles Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title | Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title_full | Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title_fullStr | Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title_full_unstemmed | Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title_short | Challenges in Drug Discovery for Neurofibromatosis Type 1-Associated Low-Grade Glioma |
title_sort | challenges in drug discovery for neurofibromatosis type 1-associated low-grade glioma |
topic | Oncology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5167692/ https://www.ncbi.nlm.nih.gov/pubmed/28066715 http://dx.doi.org/10.3389/fonc.2016.00259 |
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