Table 2. Genome edited NHPs.
Only mutant NHPs with relevant-phenotypes are shown. RTT: Rett Syndrome, PMS: Phelan-McDermid Syndrome, AHC-HH: Adrenal hypoplasia congenital and hypogonadotropic hypogonadism, ADPKD: Autosomal dominant polycystic kidney disease, PD: Parkinson’s disease, DMD: Duchenne muscular dystrophy, SCID: Severe combined immunodeficiency.
Diseases | Species | Genes | Mutations | Phenotypes | Refs |
---|---|---|---|---|---|
RTT | Cynomolgus | MECP2 | KO TALEN | RTT-like symptoms in het females | [12] |
PMS | Cynomolgus | SHANK3 | KO CRISPR | PMS-like symptoms in homo and het monkeys | [24,26] |
Longevity | Cynomolgus | SIRT6 | KO CRISPR | Prenatal death, developmental retardation | [52] |
Microcephaly | Cynomolgus | MCPH1 | KO TALEN | Microcephaly | [73] |
Circadian rhythm | Cynomolgus | BMAL1 | KO CRISPR | Reduced sleep | [74] |
AHC-HH | Cynomolgus | DAX1 | KO CRISPR | AHC-HH-like symptoms | [75] |
ADPKD | Cynomolgus | PKD1 | KO CRISPR | Cysts formation in homo and het monkeys | [63] |
PD | Rhesus | PINK1 | KO CRISPR | Neurodegeneration | [76] |
DMD | Rhesus | DMD | KO CRISPR | Muscular degeneration | [77] |
Hypercholesterolemia | Rhesus | PCSK9 | in vivo KO Meganuclease | Reduced serum cholesterol | [78] |
SCID | Marmoset | IL2RG | KO ZFN/TALEN | Immunodeficiency | [9] |