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Saab, K.

Publications and source records attributed to Saab, K..

2 recordsLinked to original sources

Human whole epigenome modelling for clinical applications with Pleiades

Gene regulation in humans extends beyond the four-letter genetic code. DNA methylation, in particular, functions as a critical epigenetic regulator, dynamically programming cellular identity, adapting gene expression in response to environmental cues, and underpinning the onset and progression of numerous diseases. Here we present Pleiades, a series of whole-genome epigenetic foundation models spanning three sizes: 90M, 600M, and 7B parameters. Pleiades is trained upon an extensive proprietary corpus of human methylation and genomic data, totalling 1.9T tokens. We introduce alignment embeddings and stacked hierarchical attention techniques to provide precise epigenetic modelling without the need for extended context lengths. Collectively, these advances enable Pleiades to perform a diverse range of down-stream biological and clinical tasks, including genomic regulatory prediction, realistic generation of cell-free DNA fragments and fragment-level cell-type-of-origin classification, within a unified and scalable computational framework. We specifically apply Pleiades to the early detection of clinical Alzheimers disease and Parkinsons disease from plasma cell-free DNA, achieving high-accuracy detection (AUROC 0.89 for AD and 0.84 for PD) using a minimally invasive blood test. Combined with plasma pTau-217, Pleiades reaches an AUROC of 0.97 for AD, underscoring the promise of multimodal epigenomic and proteomic approaches. Using mechanistic interpretability, we ground Pleiades latent features in interpretable biological signals, relating its Alzheimers predictions to fragmentomic and epigenomic properties of cfDNA. These findings demonstrate the potential of genome-wide epigenomic modelling as a clinically translatable paradigm for diagnostics and precision medicine, though prospective validation in larger, more diverse cohorts will be required to confirm clinical utility.

genomics↗

Bypassing cisplatin resistance in Nrf2 hyperactivated head and neck cancer through effective PI3Kinase targeting

BackgroundFor patients with head and neck squamous cell carcinoma (HNSCC), failure of definitive radiation combined with cisplatin nearly universally results in death. Although hyperactivation of the Nrf2 pathway can drive radiation and cisplatin resistance along with suppressed anti-tumor immunity, treatment-refractory HNSCC tumors may retain sensitivity to targeted agents secondary to synergistic lethality with other oncogenic drivers (e.g., NOTCH1 mutations). PurposeWe evaluated the efficacy of PI3K inhibitors (PI3Ki) in bypassing Nrf2-mediated cisplatin resistance in HNSCC. MethodsWe measured transcriptomic, metabolomic and signaling changes driven by PI3Kis in cisplatin-resistant HNSCCs in vitro and tested efficacy in vivo in subcutaneous, orthotopic and metastatic xenograft models using immunodeficient and humanized murine models of HNSCC coupled with spatial transcriptomics. ResultsThe PI3K pathway is activated in Nrf2-driven cisplatin-resistant HNSCC and is suitable for blockade as demonstrated in an in vivo shRNA screen. The PI3Ki gedatolisib inhibits cisplatin-resistant HNSCC proliferation, induces G2M arrest and potentiates cisplatin effectiveness through activation of autophagy, senescence and disruption of fatty acid metabolism. Gedatolisib suppresses HNSCC tumor growth in orthotopic and metastatic settings and demonstrates profound anti-tumor activity in humanized murine models of HNSCC, coupled with a reduction in hypoxia-rich regions and reduced infiltration by regulatory T lymphocytes. ConclusionOur findings emphasize the critical role of the PI3K-AKT-mTOR pathway in cisplatin-resistant HNSCC and highlight the therapeutic potential of PI3K inhibitors. Gedatolisib induced metabolic regulation and substantial re-sensitization of resistant cells to cisplatin, positioning it as a promising candidate for combination therapies aimed at overcoming primary chemo-radiation failure in HNSCC. Statement of translational relevanceCisplatin resistance, whether intrinsic or acquired, translates to treatment failure and nearly universal death in head and neck squamous cell carcinoma (HNSCC). However, the development of effective systemic regimens for cisplatin-resistant HNSCC has not yet been successful. Here, we present, for the first time, a mechanistic, biomarker-informed strategy for effective targeting of the PI3Kinase pathway in cisplatin-resistant HNSCC with substantial anti-tumor activity in both orthotopic and metastatic models, which may be capable of bypassing or reversing cisplatin resistance in this disease.

cancer biology↗