There is a significant global population affected by ophthalmic rare diseases, with over 900 different types, most of which are hereditary blinding eye diseases. These diseases have complex pathogenic mechanisms and clinical characteristics, making it challenging to achieve effective treatment using traditional approaches. However, gene therapy drugs offer a promising solution to this dilemma. Cyagen has developed a range of gene-editing models for ophthalmic diseases, including gene knockout, knock-in, and point mutation models. We also provide customization and collaborative development options tailored to the specific requirements of researchers.
Disease Name | Target Gene | Targeting Methods | Ordering |
Retinitis pigmentosa, RP | Rho | KO, CKO, Humanization(WT, Mu) | Order Now |
Mertk | KO, CKO | Order Now | |
Crb1 | KO | Order Now | |
Rd1(Pde6b) | KO, MU | Order Now | |
Rd10(Pde6b) | MU | Order Now | |
RP2 | KO, CKO | Order Now | |
Abca4 | Humanization(WT, Mu) | Order Now | |
Rpgr | KO | Order Now | |
Leber's congenital amaurosis 2 | Rpe65 | KO, MU | Order Now |
Leber's congenital amaurosis 4 | Aipl1 | KO | Order Now |
Leber's congenital amaurosis 10 | Cep290 | Humanization(WT, Mu) | Order Now |
Leber's congenital amaurosis 13 | Rdh12 | KO | Order Now |
Ocular albinism, OA | Gpr143 | KO | Order Now |
Bietti crystalline comeoretinal dystrophy, BCD | Cyp4v3 | KO | Order Now |
Corneal dystrophy, CD | Tgfbi | Mu | Order Now |
Choroideremia, CHM | Chm | CKO | Order Now |
Achromatopsia | Cngb3 | KO | Order Now |
X-linked retinoschisis, RS | Rs1 | KO | Order Now |