Sijin Luozhong

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.

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.