Organoid Models Reveal Potential Treatment for Deadly Rare Ovarian Cancer
Journal: Science Advances
Author: Seok-Young Kim et al., The Netherlands
Scientists developed patient-derived tumor models that authentically replicate Small Cell Carcinoma of the Ovary, a rare cancer affecting young women. Drug screening identified methotrexate as highly effective at clinical doses, activating cancer cell death pathways and offering new hope for this currently untreatable disease.
Study Maps Genomic Landscape of Lung Cancer Brain Metastases
Journal: Nature Medicine
Author: Somnath Tagore et al., USA
New research reveals chromosomal instability and neural-like programs in non-small cell lung cancer brain metastases. Using multimodal single-cell analysis, scientists identified distinct cancer cell populations that pre-exist in primary tumors but significantly enrich in brain metastases.
Perturbation-Response Score Method Transforms Single-Cell Analysis
Journal: Nature Cell Biology
Author: Bicna Song et al., USA
In this study the research team developed PS framework to quantify diverse cellular responses to genetic perturbations, outperforming existing methods. The approach enables dosage analysis without titration and reveals cellular response patterns in HIV expression and pancreatic differentiation.
Novel Gene Replacement Strategy Offers Hope for Blood Disorders
Journal: Molecular Therapy
Author: Kirby A. Wallace et al., USA
Researchers developed a breakthrough CRISPR-Cas9 approach for β-hemoglobinopathies, successfully replacing the entire HBB gene through homology directed repair. The strategy uses diverged coding sequences with heterologous introns to maintain proper expression while avoiding unwanted recombination events. Testing showed 34% HDR efficiency and restored β-globin expression in cell models of both sickle cell disease and β-thalassemia, with sustained editing in long-term blood stem cells.
