Deep learning–guided engineering of SpuFz1 and rational miniaturization of ωRNA enables efficient genome editing
2026
Nature Communications
Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo Base Editing in a Patient with Alpha-1 Antitrypsin Deficiency
2026
MedRxiv
In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial
2026
Nature Medicine
Prdm12 governs an epigenetic checkpoint linking neuroimmune cross-talk to CD8+ T cell exhaustion-suppressed antitumor immunity
2025
Science Advances
Spleen-targeted NeoPol-mL242 mRNA vaccine induces robust T-cell responses in a hepatocellular carcinoma model
2025
Journal of Nanobiotechnology
In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery
2025
Nature Biomedical Engineering
Sequential Amino Acid Mutagenesis-Driven De Novo Evolution of Adenine Deaminases Enables Efficient In Vivo Base Editing in Primate
2025
bioRxiv
Base Editing Gene Therapy for Heterozygous Familial Hypercholesterolemia
2025
MedRxiv
