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
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption
2025
Molecular Therapy
In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery
2024
bioRxiv
Library-Assisted Evolution in Eukaryotic Cells Yield Adenine Base Editors with Enhanced Editing Specificity
2024
Advanced Science
Therapeutic adenine base editing of human hematopoietic stem cells
2023
Nature Communications
CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric β(0)/β(0) transfusion-dependent β-thalassemia
2022
Nature Medicine
Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL
2022
Nature
