Table 1.
Delivery methods for CRISPR/Cas system in mammals
| Model-target tissue (disease) | Delivery methods | Editing gene | References | |
|---|---|---|---|---|
| Viral delivery system | No viral delivery system | |||
| Mouse blood (myeloid malignancy) | Lentivirus | Tet2, Runx1, Dnmt3a, Ezh2, Nf1, Smc3, p53 and Asxl1 | [46] | |
| Mouse lung (lung cancer) | Lentivirus | Pten and Nkx2-1 | [47] | |
| Mouse liver (NASH) | Adenovirus | Pten | [56] | |
| Mouse liver (cardiovascular disease) | Adenovirus | Pcsk9 | [57] | |
| Mouse lung (lung cancer) | Adenovirus | Eml4 and Alk | [58] | |
| Mouse brain | AAV | Mecp2, Dnmt1, Dnmt3a and Dnmt3b | [62] | |
| Mouse eye (LCA) | AAV | Yfp | [63] | |
| Mouse muscle (DMD) | AAV | Dmd | [68, 69] | |
| Mouse lung (lung cancer) | AAV | Kras, p53 and Lkb1 | [74] | |
| Mouse liver (OTC deficiency) | AAV | Otc | [72] | |
| Mouse liver (cardiovascular disease) | AAV | Pcsk9 | [78] | |
| Mouse brain (Huntington disease) | AAV | Htt | [192] | |
| Mouse brain (GBM) | AAV | Trp53, Nf1 and Rb1 | [76] | |
| Mouse brain | AAV | Camk2a, Erk2 and Actb | [193] | |
| Mouse muscle (DMD) | AAV | Dmd | [67] | |
| Mouse muscle (MDC1A) | AAV | Lama2 | [194] | |
| Mouse eye (retinal degeneration) | AAV | Nrl | [64] | |
| Mouse spleen, lungs, heart, colon, and brain (HIV/AIDS) | AAV | HIV-1 provial DNA | [195] | |
| Mouse liver (Hemophilia B) | AAV | F9 | [196] | |
| Mouse liver (cancer) | AAV | Tsgs | [77] | |
| Mouse liver | AAV | HBV cccDNA | [197] | |
| Mouse eye (X-linked retinitis pigmentosa) | AAV | Rpgr | [65] | |
| Mouse liver (tyrosinemia) | AAV | Fah | [198] | |
| Mouse liver (OTC deficiency) | AAV | Otc | [71] | |
| Mouse muscle (DMD) | AAV | Dmd | [70] | |
| Mouse brain (schizophrenia) | AAV | Mir137 | [75] | |
| Mouse muscle (ALS) | AAV | Igf1 | [66] | |
| Mouse liver (tyrosinemia) | Plasmid | Fah | [84] | |
| Mouse liver | Plasmid | HBsAg | [85] | |
| Rat eye (retinitis pigmentosa) | Plasmid | (Rho(S334)) | [86] | |
| Mouse eye (retinitis pigmentosa) | Plasmid | Rho | [87] | |
| Mouse brain (MB and GBM) | Plasmid | Trp53, Pten and Nf1 | [199] | |
| Mouse brain | Plasmid | Satbs | [89] | |
| Mouse eye (IRDs) | Plasmid | Rho-P23H | [88] | |
| Mouse liver (tyrosinemia) | mRNA | Fah | [90] | |
| Mouse liver (cardiovascular disease) | mRNA | Pcsk9 | [91, 93] | |
| Mouse liver (tyrosinemia) | mRNA | Ttr | [92] | |
| Mouse liver, kidney, and lung | mRNA | floxed tdTomato | [200] | |
| Mouse ear | Protein | Egfp | [94] | |
| Mouse muscle (DMD) | Protein | Dmd | [100] | |
| Mouse brain (FXS) | Protein | Grm5 | [101] | |
| Mouse brain (Alzheimer’s disease) | Protein | Th, Bace1 | [96] | |
| Mouse liver and spleen | Protein | Pten | [99] | |
| Mouse liver and spleen (HT1) | Protein | Hpd | [95] | |
AAV adeno-associated virus, ALS amyotrophic lateral sclerosis, DMD Duchenne muscular dystrophy, FXS fragile X syndrome, GBM glioblastoma, HIV/AIDS human immunodeficiency virus/acquired immunodeficiency syndrome, HT1 hereditary tyrosinemia type I, IRDs inherited retinal degenerations, LCA Leber congenital amaurosis, MB medulloblastoma, MDC1A congenital muscular dystrophy type 1A, NASH non-alcoholic steatohepatitis, OTC ornithine transcarbamylase