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Disease Model Pipeline

Indication

Model

Gene Targeting

Founders

Characterization

 

Heart Disease

ExeGen® LDLR-/-&+/-

     
 

Cystic Fibrosis

ExeGen® CFTR-/-

     
 
 

ExeGen® CFTRF508del/F508del

     
 

Cardiac Arrhythmia

ExeGen® SCN5A+/E558X

     
 

Cancer

ExeGen® TP53+/R175H, R175H/R175H

     
 
 

ExeGen® TP53+/R175H& KRAS+G12D

    In Progress
 

Neuromuscular

ExeGen® DMD+/ex52del

     
 

Neurodegeneration

ExeGen® ATM-/-

     
 
 

ExeGen® CLN3ex7&8del/ex7&8del

    In Progress
 
 

ExeGen® HTT+Ex1+(CAG)85

    In Progress
 

Hypertrophic Cardiomyopathy

ExeGen® MYH7+/R403Q

    In Progress
 

Nervous System

ExeGen® NF-1+/-

    In Progress
 
Renal Disease

ExeGen® PKD1+/-

    In Progress
 
 

ExeGen® PKD1+/R3277C

    In Progress
 
Blood Disorders

ExeGen® SCDhHBA/hHBA/hHBG-hHBBS/ hHBG-hHBBS