ArticleThe Journal of clinical investigation2021
Humanized neurofibroma model from induced pluripotent stem cells delineates tumor pathogenesis and developmental origins.
Article in The Journal of clinical investigation, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 45 papers.
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Who cites it
45 citing papers in PubMed, 73 citations in OpenAlex.
- Advancing neurofibromatosis research: a comparative review of animal models and their utility in dissecting the tumor microenvironment.Laboratory animal research · 2026Review
- Proof-of-principle of NF1 gene therapy in plexiform neurofibroma xenograft mouse models.Communications biology · 2026Article
- A standardized imaging and analysis workflow for quantitative evaluation of cutaneous neurofibromas in Nf1-KO mice.PloS one · 2026Article
- Neurofunctional MOF nanoparticles integrated with extracellular matrix hydrogel for neuro-Vascularized bone regeneration.Materials today. Bio · 2025Article
- Development of an adeno-associated virus vector for gene replacement therapy of NF1-related tumors.Nature communications · 2025Article
- Dermal fibroblast-derived extracellular matrix (ECM) synergizes with keratinocytes in promoting re-epithelization and scarless healing of skin wounds: Towards optimized skin tissue engineering.Bioactive materials · 2025Article
- Biomaterials-mediated biomineralization for tumor blockade therapy.Nanomedicine (London, England) · 2025Review
- Neurofibroma of bladder: A case report and literature review.Urology case reports · 2025Article
- Disulfiram and cancer immunotherapy: Advanced nano-delivery systems and potential therapeutic strategies.International journal of pharmaceutics: X · 2024Review
- Disrupting CENP-N mediated SEPT9 methylation as a strategy to inhibit aerobic glycolysis and liver metastasis in colorectal cancer.Clinical & experimental metastasis · 2024Article
- The pan-cancer landscape of crosstalk between leukocyte transendothelial migration-related genes and tumor microenvironment relevant to prognosis and immunotherapy response.Translational cancer research · 2024Article
- Unraveling neuronal and metabolic alterations in neurofibromatosis type 1.Journal of neurodevelopmental disorders · 2024Review
- Unveiling the role of AGT in lipid metabolism and regulated cell death in colon cancer.Neoplasia (New York, N.Y.) · 2024Article
- TNC upregulation promotes glioma tumourigenesis through TDG-mediated active DNA demethylation.Cell death discovery · 2024Article
- A platform for rapid patient-derived cutaneous neurofibroma organoid establishment and screening.Cell reports methods · 2024Article
- The Role and Mechanism ofMolecules (Basel, Switzerland) · 2024Review
- TheCancers · 2024Review
- Stem cell modeling of nervous system tumors.Disease models & mechanisms · 2024Review
- Advances and prospects of precision nanomedicine in personalized tumor theranostics.Frontiers in cell and developmental biology · 2024Review
- Article
Corrections and comments
- Erratum issued
Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
Neurofibromatosis type 1 (NF1) is a common tumor predisposition syndrome caused by NF1 gene mutation, in which affected patients develop Schwann cell lineage peripheral nerve sheath tumors (neurofibromas). To investigate human neurofibroma pathogenesis, we differentiated a series of isogenic, patient-specific NF1-mutant human induced pluripotent stem cells (hiPSCs) into Schwannian lineage cells (SLCs). We found that, although WT and heterozygous NF1-mutant hiPSCs-SLCs did not form tumors following mouse sciatic nerve implantation, NF1-null SLCs formed bona fide neurofibromas with high levels of SOX10 expression. To confirm that SOX10+ SLCs contained the cells of origin for neurofibromas, both Nf1 alleles were inactivated in mouse Sox10+ cells, leading to classic nodular cutaneous and plexiform neurofibroma formation that completely recapitulated their human counterparts. Moreover, we discovered that NF1 loss impaired Schwann cell differentiation by inducing a persistent stem-like state to expand the pool of progenitors required to initiate tumor formation, indicating that, in addition to regulating MAPK-mediated cell growth, NF1 loss also altered Schwann cell differentiation to promote neurofibroma development. Taken together, we established a complementary humanized neurofibroma explant and, to our knowledge, first-in-kind genetically engineered nodular cutaneous neurofibroma mouse models that delineate neurofibroma pathogenesis amenable to future therapeutic target discovery and evaluation.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.