TABLE 2.

CRISPR clinical trials for some inherited disorders recorded in the current review.

Disease	Target gene	Model	Findings	References
β-thalassemia	BCL11A	patients with TDT and SCD	▪ Patients had high levels of allelic editing in	Frangoul et al. (2021)
			▪ bone marrow and blood, increases in fetal hemoglobin that were distributed pancellularly, transfusion independence, and (in the patient with SCD) elimination of vaso-occlusive episodes
Recessive dystrophic epidermolysis bullosa	COL7A1	patient keratinocytes	▪ This ex vivo non-viral approach rendered a large proportion of corrected cells producing a functional collagen VII variant	Bonafont et al. (2019)
			▪ The effective targeting of the epidermal stem cell population enabled long-term regeneration of a properly adhesive skin upon grafting onto immunodeficient mice
			▪ A safety assessment by next-generation sequencing (NGS) analysis of potential off-target sites did not reveal any unintended nuclease activity
Duchenne muscular dystrophy (DMD)	dmd	human patient with DMD–derived iPSCs	▪ Correction of exon 44 deletions through exon skipping or reframing of surrounding exons could potentially treat ∼12% of patients with DMD	Min et al. (2019)