Disease Model Pipeline

Indication

Model

Gene Targeting

Founders

Characterization

Validation

 

Heart Disease

ExeGen® LDLR-/-&+/-

       
 

Cystic Fibrosis

ExeGen® CFTR-/-

       
 
 

ExeGen® CFTRF508del/F508del

       
 

Cardiac Arrhythmia

ExeGen® SCN5AE555X

       
 

Cancer

ExeGen® TP53+/R167H, R167H/R167H

       
 
 

ExeGen® TP53+/R167H& KRAS+/G12D

       
 

Neuromuscular

ExeGen® DMD+/ex52del

       
 

Neurodegeneration

ExeGen® ATM-/-

       
 
 

ExeGen® CLN3ex7&8del/ex7&8del

       
 
 

ExeGen® HTT+/Ex1+(CAG)n

       
 

Nervous System

ExeGen® NF-1+/-

       
 
Renal Disease

ExeGen® PKD mutant