Low Efficiency of Homology-Independent Targeted Integration for CRISPR/Cas9 Correction in the Vicinity of SLC26A4 c.919–2A>G Variant
Efficiency of genome editing using modified single-stranded oligodeoxyribonucleotides in human cells
Engineering TadA ortholog-derived cytosine base editor without motif preference and adenosine activity limitation
Application of an artificial deaminase system for restoration of mutated mRNAs in the macular mouse caused by T>C mutation
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system
Reprogramming human B cells with custom heavy-chain antibodies
Targeted gene addition at the CX3CR1 locus of human HSPCs improves their ability to repopulate the myeloid CNS compartment and drives lineage specific and robust transgene expression in their CNS progeny
Combination of engineered FnCas9 and extended gRNAs for PAM-flexible, robust and nucleobase specific editing and diagnostics
Bioengineering novel AAV9-mGULO-GT for multi-disease gene therapy: Targeting mutated GULO expression to cure scurvy and brain diseases.
Enhancing Precise Genome Editing in Human Pluripotent Stem Cells through Dual Inhibition of DNA Damage Response and Repair Pathways