PDGFRA (Platelet Derived Growth Factor Receptor Alpha)

The patient has been under observation without any further intervention for 70 months since diagnosis, with no evidence of disease recurrence. In addition to the rarity of cLNC, this is a unique case in terms of bifocality, anaplastic histology, and the described genetic abnormalities.

Results highlight how targeted enrichment and maturation of specific progenitor subpopulations within hBMSCs allows rapid induction of desired cell types. These results place hBMSCs as a robust source of OPCs, unlocking the possibility for cell transplantation therapy for myelin deficiency in the central nervous system.

Similarly, genes related to inflammation, such as Tnfa (tumour necrosis factor α), Il-6 (interleukin 6), Tlr4 (Toll-like receptor 4) and Tlr2 (Toll-like receptor 2), were unaffected. In conclusion, these findings indicate that short-term lipid exposure alters gene expression patterns without measurable structural changes, suggesting that transcriptional responses may precede overt morphological remodelling in fetal skeletal muscle.

The remarkable success of tyrosine kinase inhibitors such as imatinib (IM) in treating GIST has established them as a paradigm of precision medicine in modern oncology...Emerging evidence suggests that analogous non-genetic persistence states also exist in GIST, including dormant cells and KITlow stem-like/CSC-like subpopulations. This review summarizes the fundamental regulatory mechanisms of tumor dormancy and CSC biology, discusses their candidate manifestations in GIST, and proposes innovative therapeutic strategies based on these insights.

P1/2, N=94, Recruiting, Deciphera Pharmaceuticals, LLC | Not yet recruiting --> Recruiting

Our study develops a novel prognostic model IPCDS for OV. IPCD-related gene PDGFRA serves as a prognosis factor play in predicting survival outcomes of OVs.

Between the 1990s and early 2000s, GIST was identified as a tumor characterized by KIT or PDGFRA mutations, resulting in imatinib being established as an effective targeted therapy...These inconsistencies have hindered pathologic reporting and communication between pathologists and clinicians. This review comprehensively overviews the historical background, molecular subtypes, and risk classification systems of GIST, focusing on evolving issues in mitotic rate evaluation and the application of risk classification systems in clinical practice.

P2, N=48, Recruiting, Asan Medical Center | Trial completion date: Dec 2026 --> Dec 2027 | Trial primary completion date: Aug 2026 --> Aug 2027

P2, N=28, Recruiting, Asan Medical Center | Not yet recruiting --> Recruiting

Resistance to drugs in sarcoma is multifactorial and changeable. Nevertheless, fusing mechanistic knowledge with biomarker - guided combination/sequential therapies offers a clear way to improve patient situations.

Since the introduction of imatinib in the early 21st century, the management of metastatic GIST has shifted from solely surgical intervention to a systemic, chronic disease management model centered on tyrosine kinase inhibitors (TKIs)...These challenges underscore the necessity of this review, which discusses current standard drug treatment strategies for advanced GIST, including sequential TKIs therapy and investigations into mechanisms of drug resistance. Finally, the review explores precise and actionable future directions for GIST drug development and clinical management, including mutation-stratified therapeutic sequencing, rational TKI-based combination regimens, and circulating tumor DNA (ctDNA)-guided real-time treatment monitoring and resistance surveillance.

Intranasal IL-4 delivery may represent a promising therapeutic strategy for enhancing white matter integrity in leukodystrophies and MS. Abbreviations: MS, multiple sclerosis; IL-4, Interleukin 4; CNS, central nervous system; OPCs, oligodendrocytes precursor cells; IL-4R, IL-4 receptor; i.n., intranasal; LPC, lysophosphatidylcholine; PVL, periventricular leukomalacia; EAE, experimental autoimmune encephalomyelitis; dpi, days post immunization; dpl, days post lesion; Nkx2.2, NK2 homeobox 2; Olig2, oligodendrocyte transcription factor 2; OLs, oligodendrocytes; SOX10, SRY-Box Transcription Factor 10; PDGFRα, platelet derived growth factor receptor alpha; CNPase, 2',3'-Cyclic Nucleotide 3' Phosphodiesterase; APC, adenomatous polyposis coli; ASPA, aspartoacylase; GFAP, glial fibrillary acidic protein; Iba1, ionized calcium binding adaptor molecule 1; MBP, myelin basic protein; TEM, transmission electron microscopy; CC, corpus callosum; GCC, genu of the corpus callosum; CG, cingulum; CTX, cortex; STR, striatum; EC, external capsule; Pre-OLs, pre-myelinating oligodendrocytes; STAT6, signal transducer and activator of transcription 6; Pio, pioglitazone; T3, triiodothyronine; CNTF, ciliary neurotrophic factor; PPARγ, peroxisome proliferator-activated receptor γ.