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. Author manuscript; available in PMC: 2021 Mar 13.
Published in final edited form as: Curr Opin Genet Dev. 2020 Jul 18;65:160–168. doi: 10.1016/j.gde.2020.05.040

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]