CRISPR-Cas9 base editing corrects sickle cell disease mutation in human hematopoietic stem cells
Clinical trial results demonstrate safe and effective gene editing for sickle cell disease treatment with long-term engraftment.
查看全文2026年03月31日
数据来源:bioRxiv · Nature · PubMed · Cell · Science
Clinical trial results demonstrate safe and effective gene editing for sickle cell disease treatment with long-term engraftment.
查看全文Novel lipid nanoparticle formulation enables efficient mRNA delivery to tumor microenvironment, showing promising results in phase 2 trial.
查看全文Comprehensive map reveals new cell types and developmental trajectories, providing insights into neurodevelopmental disorders.
查看全文Cryo-EM structures reveal molecular mechanisms of antibody evasion and inform next-generation vaccine design.
查看全文Specific gut bacteria strains identified as key predictors of checkpoint inhibitor efficacy in melanoma patients.
查看全文Machine learning-guided enzyme engineering achieves 100x improvement in PET degradation efficiency.
查看全文CD19-targeted CAR-T cells show durable responses in severe lupus and rheumatoid arthritis.
查看全文Patient-derived organoids capture tumor evolution and identify novel therapeutic targets.
查看全文High-resolution spatial profiling reveals immunosuppressive niches and potential combination therapy targets.
查看全文AAV-mediated gene delivery shows sustained improvement in visual function with minimal adverse events.
查看全文