Direct conversion of HD patient fibroblasts into neuron-like cells.

Human fibroblasts derived from a normal individual (16Q) or HD patients (68Q and 86Q) were infected with lentiviral shRNAs against human PTB. Nineteen days following the treatment, cells were used for analyses. (A) Immunostaining of the trans-differentiated cells with a neuron-specific marker, Tuj1, antibody. Arrow heads indicate thin or short neurites and arrows show broken neurites. Scale bar, 10 µm. Cell counting results showing the percentage of cells with neuron-like morphology positive in Tuj1 staining (B), with abnormal neurites (C), or with degenerated (breakdown) neurites (D). Data are shown as mean ± SD; n = 3. *p<0.05.