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
Therapeutic base editing of human hematopoietic stem cells
2020
Nature Medicine
