Bowen Li

Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells.

Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for...

Lipid nanoparticles optimized for large RNA cargo and tissue targeting enhance in vivo genome editing.

Lipid Nanoparticles Based Multi-Scale Systemic Immune Programming for Cancer Therapy.

A dual-track therapeutic paradigm that integrates tumor-directed immune activation with host physiological restoration is proposed, which positions LNP-based therapies to treat cancer as a systemic immune-metabolic disorder rather than a localized disease.

Nonviral delivery of chemically modified tRNA rescues nonsense mutations in cystic fibrosis.

This work combined site-specific chemical modification of sup-tRNAs with cargo-tailored pulmonary lipid nanoparticle (LNP) engineering to overcome barriers and restore cystic fibrosis transmembrane conductance regulator (CFTR) expression and function.

Locus-specific transposable element expression drives human hematopoietic stem cell disease pathophysiology

This study provides direct evidence that VEXAS-specific TE govern HSC clonal dominance, thereby uncovering a regulatory axis underlying HSC biology and disease mechanisms, opening a therapeutic strategy directed towards the repetitive genome.