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To date, a plenty of techniques for the detection of JAK2V617F is used over different laboratories, with substantial differences in specificity and sensitivity. Therefore, to provide reliable and comparable results, the standardization of molecular techniques is mandatory.A network of 19 centers was established to 1) evaluate the inter- and intra-laboratory variability in JAK2V617F quantification, 2) identify the most robust assay for the standardization of the molecular test and 3) allow consistent interpretation of individual patient analysis results. The study was conceived in 3 different rounds, in which all centers had to blindly test DNA samples with different JAK2V617F allele burden (AB) using both quantitative and qualitative assays.The positivity of samples with an AB < 1% was not detected by qualitative assays. Conversely, laboratories performing the quantitative approach were able to determine the expected JAK2V617F AB. Quantitative results were reliable across all mutation loads with moderate variability at low AB (0.1 and 1%; CV = 0.46 and 0.77, respectively). Remarkably, all laboratories clearly distinguished between the 0.1 and 1% mutated samples.In conclusion, a qualitative approach is not sensitive enough to detect the JAK2V617F mutation, especially at low AB. On the contrary, the ipsogen JAK2 MutaQuant CE-IVD kit resulted in a high, efficient and sensitive quantification detection of all mutation loads. This study sets the basis for the standardization of molecular techniques for JAK2V617F determination, which will require the employment of approved operating procedures and the use of certificated standards, such as the recent WHO 1st International Reference Panel for Genomic JAK2V617F.
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http://dx.doi.org/10.18632/oncotarget.15940 | DOI Listing |
Alzheimers Dement
September 2025
Department of Neurology, Beijing TianTan Hospital, Capital Medical University, Beijing, China.
Cognitive impairment and dementia, including Alzheimer's disease (AD), pose a global health crisis, necessitating non-invasive biomarkers for early detection. This review highlights the retina, an accessible extension of the central nervous system (CNS), as a window to cerebral pathology through structural, functional, and molecular alterations. By synthesizing interdisciplinary evidence, we identify retinal biomarkers as promising tools for early diagnosis and risk stratification.
View Article and Find Full Text PDFInt J Dermatol
September 2025
Dermatology Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.
Introduction: Cutaneous scalp metastases from breast carcinoma (CMBC) represent an uncommon manifestation of metastatic disease, with heterogeneous clinical presentations, including nodular or infiltrative lesions and scarring alopecia (alopecia neoplastica). The absence of standardized diagnostic criteria, particularly for alopecic phenotypes, poses challenges to early recognition of CMBC, which may represent either the first indication of neoplastic progression or a late recurrence.
Materials And Methods: We retrospectively analyzed a multicenter cohort of 15 patients with histologically confirmed CMBC.
Mol Ther Methods Clin Dev
June 2025
Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Pompe disease is a glycogen storage disorder caused by mutations in the acid α-glucosidase (GAA) gene, leading to reduced GAA activity and glycogen accumulation in heart and skeletal muscles. Enzyme replacement therapy with recombinant GAA, the standard of care for Pompe disease, is limited by poor skeletal muscle distribution and immune responses after repeated administrations. The expression of GAA in muscle with adeno-associated virus (AAV) vectors has shown limitations, mainly the low targeting efficiency and immune responses to the transgene.
View Article and Find Full Text PDFComput Struct Biotechnol J
August 2025
Institut de Recherche en Cancérologie de Montpellier (IRCM), Équipe Labellisée Ligue Contre le Cancer, INSERM U1194, Université de Montpellier, Institut régional du Cancer de Montpellier (ICM), Montpellier, France.
Digital twins (DTs) are emerging tools for simulating and optimizing therapeutic protocols in personalized nuclear medicine. In this paper, we present a modular pipeline for constructing patient-specific DTs aimed at assessing and improving dosimetry protocols in PRRT such as therapy. The pipeline integrates three components: (i) an anatomical DT, generated by registering patient CT scans with an anthropomorphic model; (ii) a functional DT, based on a physiologically-based pharmacokinetic (PBPK) model created in SimBiology; and (iii) a virtual clinical trial module using GATE to simulate particle transport, image simulation, and absorbed dose distribution.
View Article and Find Full Text PDFMater Today Bio
October 2025
School of Pharmacy, Henan Medical University, Xinxiang, Henan, China.
Breast cancer continues to present a major clinical hurdle, largely attributable to its aggressive metastatic behavior and the suboptimal efficacy of standard chemotherapeutic regimens. Cisplatin (CDDP) is a representative platinum drug in the treatment of breast cancer, however, its therapeutic application is often constrained by systemic toxicity and the frequent onset of chemoresistance. Here, we introduce a novel charge-adaptive nanoprodrug system, referred to as PP@, engineered to respond to tumor-specific conditions.
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