CRISPR J 2021 Apr;4(2):207-222
University Children's Hospital, Department of Pediatrics I, Hematology and Oncology, University of Tübingen, Germany; University of Tübingen, Tübingen, Germany.
Mutations in the human gene are the cause of β-hemoglobinopathies, one of the most common inherited single-gene blood disorders in the world. Novel therapeutic approaches are based on lentiviral vectors (LVs) or CRISPR-Cas9-mediated gene disruption to express adult hemoglobin (HbA), or to reactivate the completely functional fetal hemoglobin, respectively. Nonetheless, LVs present a risk of insertional mutagenesis, while gene-disrupting transcription factors (BCL11A, KLF1) involved in the fetal-to-adult hemoglobin switch might generate dysregulation of other cellular processes. Read More