Epitope editing of KIT enables antibody-based, non-genotoxic conditioning that selectively enriches therapeutic BCL11A-edited haematopoietic stem/progenitor cells, supports durable engraftment, preserves clonal diversity and enhances induction of fetal haemog…
Sleeping Beauty transgene overexpression
Bidirectional Sleeping Beauty plasmids encoding codon-optimized cDNA of the gene of interest (KIT) or its mutated variants were cloned downstream of an EF1 p… [+39149 chars]
Bidirectional Sleeping Beauty plasmids encoding codon-optimized cDNA of the gene of interest (KIT) or its mutated variants were cloned downstream of an EF1 p… [+39149 chars]