Disease Name | Target Genes | Targeting Methods | Ordering |
Achondroplasis, ACH | Fgfr3 | Mu | Order Now |
Adrenoleukodystrophy, ALD | Abcd1 | KO | Order Now |
Aicardi-Goutieres syndrome, AGS | Adar | KO | Order Now |
Alport's syndrome, AS | Col4a5 | Mu | Order Now |
Alström syndrome | Alms1 | Mu | Order Now |
Autosomal dominant nonsyndromic deafness | Tmc1 | Mu | Order Now |
Chronic granulomatous disease, CGD | Cybb | KO | Order Now |
Congenital nephrogenic dia-betesinsipidus, CNDI | Avpr2 | Mu | Order Now |
Dilated cardiomyopathy, DCM | Bag3 | Mu | Order Now |
Epidermolysis bullosa, EB | Krt14 | Mu | Order Now |
Col7a1 | Humanization(WT, Mu) | Order Now | |
Familial mediterranean fever, FMF | Mefv | KI | Order Now |
Fragile X syndrome, FXS | Fmr1 | KO | Order Now |
Hereditary multi-infarct dementia, hMID | Notch3 | Mu | Order Now |
Hereditary spastic paraplegia, HSP | Spast | TG | Order Now |
Hutchinson-Gilford progeria syndrome, HGPS | Lmna | Mu | Order Now |
Zmpste24 | KO | Order Now | |
Hypertrophic cardiomyopathy, HCM | Myh7 | Mu | Order Now |
Hyperuricemia | Uox | KO | Order Now |
Hypophosphatasia, HPP | Alpl | KO | Order Now |
Hypophosphatemic rickets | Dmp1 | KO | Order Now |
IgG4-related disease, IgG4-RD | Lat | Mu | Order Now |
Kallmann syndrome, KS | Sox10 | KO | Order Now |
Laron syndrome | Ghr | KO | Order Now |
Marfan syndrome | Fbn1 | Mu | Order Now |
Melanoma | Braf | CKO | Order Now |
Mucopolysaccharide diseases, MPS | Ids | KO | Order Now |
Idua | KO | Order Now | |
Nephrotic syndrome, NS | Nphs2 | Mu | Order Now |
Noonan syndrome, NS | Raf1 | Mu | Order Now |
Osteogenesis imperfecta, OI | Bmp1 | CKO | Order Now |
Prader-Willi syndrome, PWS | Snrpn | KO | Order Now |
Primary carnitine deficiency, PCD | Slc22a5 | Mu | Order Now |
PRKAG2 cardiac syndrome | Prkag2 | Mu | Order Now |
Severe combined immunodificiency, SCID | Il2rg | KO | Order Now |
Simple 46, XY gonadal dysgenesis syndrome | Nr5a1 | CKO | Order Now |
Transthyretin amyloidosis, ATTR | Ttr | Humanization(WT, Mu) | Order Now |
Wiskott-Aldrich syndrome, WAS | Wasp | KO | Order Now |