What are the options for gene editing therapy for hemophilia?

The methods of hemophilia gene editing treatment mainly include in vivo gene editing and in vitro gene editing. 1. In vivo gene editing: the most ideal way to treat hemophilia is to correct or replace mutated clotting factor genes at the genomic level. The introduction of traditional gene therapy tools into the field of gene editing has made in vivo editing at the chromosome level possible. However, the safety and accuracy of this technology are still important challenges. 2. In vitro gene editing: transplantation of corrected autologous cells avoids immune rejection and enables genotypic and phenotypic analysis prior to transplantation. For example, patient-specific induced pluripotent stem cells, which have unlimited self-renewal capacity, can be derived from single-cell clones and can differentiate into different types of cells in the body.