PRJNA433002
Picking the right target for HSC-mediated gene therapy
Hematopoietic stem cell (HSC)-mediated gene therapy holds enormous potential for the treatment of hematological diseases and disorders. However, most strategies target CD34+ cells, a heterogeneous mix of predominantly committed progenitors and only very few multipotent HSCs. Consequently, the HSC targeting efficiency is low, the long-term outcome poor and currently available approaches very costly.