Title: | Disease modeling and lentiviral gene transfer in patient-specific induced pluripotent stem cells from late-onset Pompe disease patient |
Journal : | Molecular therapy. Methods & clinical development, 2, 15023 |
Authors: | Sato Y ; Kobayashi H ; Higuchi T ; Shimada Y ; Era T ; Kimura S ; Eto Y ; Ida H ; Ohashi T |
Material Type: | Article |
Publication Date: | 2015 |
Size: | p. 1-8 |
Languages: | English |
Keywords : | CRISPR/Cas9 ; cross-sectional study ; late-onset Pompe disease ; restriction enzyme |