Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy

R Eggenschwiler, K Loya, G Wu, A D Sharma, M Sgodda, D Zychlinski, C Herr, D Steinemann, J Teckman, R Bals, M Ott, A Schambach, H R Scholer, T Cantz