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Evaluating CAR-T cells from neurofibromatosis type 1 (NF1) patients for targeting AXL in malignant peripheral nerve sheath tumors associated with NF1.
Br J Cancer
September 2025
Department of Neurology, National Taiwan University Hospital, Taipei, Taiwan.
Background: Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder characterized by neurofibromas, with 5-13% of patients risk developing malignant peripheral nerve sheath tumors (MPNST). Current treatments for MPNST are largely ineffective. AXL, overexpressed in MPNST, is a potential target for Chimeric Antigen Receptor T (CAR-T) cell therapy.
View Article and Find Full Text PDFCase Report: Malignant transformation of ganglioneuroma in the rectovaginal septum to malignant peripheral nerve sheath tumor based on neurofibromas.
Front Oncol
June 2025
Department of Obstetrics and Gynecology, Third Xiangya Hospital, Central South University, Changsha, China.
Malignant peripheral nerve sheath tumor (MPNST) is a type of soft tissue sarcoma that commonly occurs in the trunk, limbs, and head and neck regions, but rarely in the pelvic area. Nearly half of MPNST cases are secondary to neurofibromatosis type 1 (NF1). This case report discusses a 29-year-old woman diagnosed with NF1 and pseudohermaphroditism, who presented with a large mass in the rectovaginal septum.
View Article and Find Full Text PDFThe RXR Agonist MSU-42011 Reduces Tumor Burden in a Murine Preclinical NF1-Deficient Model.
Cancers (Basel)
June 2025
Department of Pharmacology and Toxicology, Michigan State University, East Lansing, MI 48824, USA.
Neurofibromatosis type 1 (NF1) is a prevalent inherited disorder, with approximately 50% of affected individuals developing plexiform neurofibromas (PNFs), which can progress to highly aggressive malignant peripheral nerve sheath tumors (MPNSTs). While selumetinib is FDA-approved for PNFs, its efficacy in MPNSTs is limited and associated with dose-limiting toxicities. deficiency drives tumorigenesis and alters immune dynamics via RAS hyperactivation.
View Article and Find Full Text PDFCross-Species Functional Genomic Screens Identify Novel Therapeutic Targets in Malignant Peripheral Nerve Sheath Tumors.
Am J Pathol
June 2025
Departments of Pathology and Laboratory Medicine, Medical University of South Carolina, Charleston, South Carolina. Electronic address:
As oncogenic pathways are highly conserved in vertebrates, genetically engineered mouse models can potentially be used to identify therapeutic targets relevant to rare human cancers, such as malignant peripheral nerve sheath tumors (MPNSTs). To test this, genome-scale shRNA screens designed to identify genes driving proliferation and survival were performed in five MPNST cultures derived from P-GGFβ3 mice and three human MPNST cell lines. Several hundred gene hits mediating proliferation and survival were identified in human and mouse MPNST cells, many of which have been implicated in proliferation and survival in other cancers and/or mediate the pathogenesis of other cancer types.
View Article and Find Full Text PDFMultidisciplinary collaborative treatment of a giant malignant peripheral nerve sheath tumor on the top of the head: a case report.
Discov Oncol
May 2025
Radiology Department, The First Hospital of Zhejiang University, Hangzhou, 310000, China.
Background: Malignant peripheral nerve sheath tumors (MPNSTs) rarely occur on the top of the head. MPNSTs are usually characterized by invasiveness, recurrence, and metastasis.
Case Presentation: Herein we report a 44-year-old female patient who received multidisciplinary collaborative treatment for a giant MPNST on the top of her head.