SCIE.05.149
FVIII REGULATES ENDOTHELIAL CELL FUNCTIONALITY
SCIE.11.48
DEVELOPMENT OF AN ALLELE-SPECIFIC EPIGENETIC SILENCING PLATFORM FOR THE TREATMENT OF SCA2
SCIE.11.96
DEVELOPMENT OF AN EPIGENETIC EDITING STRATEGY FOR THE TREATMENT OF HUNTINGTON'S DISEASE.
SCIE.11.189
EVALUATING THE EFFICACY OF A GENE EDITING STRATEGY FOR PROGRESSIVE FAMILIAR INTRAHEPATIC CHOLESTASIS TYPE 2 (PFIC-2)