Eggenschwiler R, Loya K, Wu G, Sharma ADeep, Sgodda M, Zychlinski D, Herr C, Steinemann D, Teckman J, Bals R, et al. Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy. Stem Cells Transl Med. 2013.