Table 3.

CRISPR–Cas applications in selected monogenic diseases.

Disease	Target	Animal Model	Delivery System	Strategy	References
Leber congenital amaurosis type 10 (LCA10)	CEP290	HuCEP290 IVS26 KI mouse eye	Adeno-Associated Virus (AAV); (subretinal injection)	Non-homologous end joining (NHEJ) mediated aberrant splicing	[79]
Duchenne muscular dystrophy (DMD)	Dmd	mdx mice muscle	AAV; intramuscular injection	NHEJ mediated mutant exon 23 skipping	[80]
Sickle cell disease (SCD)	BCL11A erythroid enhancer	CD34+ human hematopoietic stem/progenitor cells (HSPCs) from sickle cell disease patient	Ribonucleoprotein (RNP); electroporation	NHEJ mediated enhancer disruption	[81]
Genetic Deafness	Tmc1	Beethoven (Bth) mouse ear	AAV; Inner ear injections	NHEJ mediated mutant Tmc allele disruption	[82]