Rare Neurological Disease Models

Neurological disorders account for 60% to 80% of rare diseases, making them one of the most complex and common groups of diseases with unknown etiology and pathogenic mechanisms. Cyagen has developed a range of gene-editing models, including gene knockout, knock-in, and point mutation models, to address neurological diseases. We also offer customization and collaborative development options to meet researchers' specific needs.


Disease Name Target Genes Targeting Methods Ordering
Spinal muscular atrophy, SMA Smn1 Humanization Order Now
Smn2 KI Order Now
Frontotemporal dementia, FD Mapt Humanization(WT, Mu) Order Now
Spinocerebellar ataxia, SCAs Atxn3 Humanization(WT, Mu) Order Now
Familial dysautonomia, FD Elp1 Humanization(WT, Mu) Order Now
Spinal muscular atrophy with respiratory distress type 1, SMARD1  Ighmbp2 Humanization(WT, Mu) Order Now
Parkinson's disease, PD Snca Humanization(WT, Mu) Order Now
Lrrk2 Mu Order Now
Amyotrophic lateral sclerosis, ALS Tardbp Humanization(WT, Mu) Order Now
Fus Humanization(WT, Mu) Order Now
Sod1 TG Order Now
Huntington's disease, HD Htt TG Order Now
Rett syndrome Mecp2 Humanization(WT, Mu) Order Now
Cornelia de Lange syndrome, CdLS Nipbl KO Order Now
Gaucher disease, GD Gba CKO Order Now
Niemaoh-Pick disease, NPD Npc1 CKO Order Now
Hereditary spastic paraplegia 2, HSP2 Plp1 CKO Order Now
Joubert syndrome, JS Ahi1 CKO Order Now
Angelman syndrome, AS Ube3a KO Order Now
Friedreich's ataxia, FRDA Fxn KO Order Now
Metachromatic leukodystrophy, MLD Arsa KO Order Now
GM1 gangliosidosis, GM1 Glb1 KO Order Now
Charcot-Marie-Tooth disease, CMT Pmp22 TG Order Now
Subacute necrotizing encephalomyelopathy, SNE Surf1 KO Order Now
Vanishing white matter disease, VWM Eif2b5 Mu Order Now