Table 1 Characteristics of human fibroblast-derived SMA induced pluripotent stem cell (iPSC) lines and controls.
iPSC line | Diagnosis | Mutations SMN1 | Sex | Age (Months) | Reprogramming strategy | Reference |
|---|---|---|---|---|---|---|
SMA 1.1 | SMA1 | EX7-8DEL | Male | 3 | Non-viral 6 factors: OSKM+LN | Corti et al. 2012 |
SMA 2.1 | SMAI | EX7-8DEL | Male | 2 | Non-viral 6 factors: OSKM+LN | Corti et al., 2012 |
SMA 3.1 | SMAI | EX7-8DEL | Male | 3 | Non-viral 6 factors: OSKM+LN | This report |
SMA 4.1 | SMAI | EX7-8DEL | Male | 3 | Viral 4 factors: OSKM | This report |
SMA 5.1 | SMA | EX7-8DEL | Female | 3 | Viral 4 factors: OSKM | This report |
HET | Father of patient 1 | Heterozygous EX7-8DEL | Male | N/Avail | Non-viral 6 factors: OSKM+LN | Corti et al. 2012 |
19.9 | Healthy Donor | – | Male | Newborn | Non-viral 6 factors: OSKM+LN | Yu et al. 2009 |
CTR AC 1.1 | Healthy Donor | – | Female | 24 | Non-viral 6 factors: OSKM+LN | Simone et al. 2014 |
CTR AC 1.2 | Healthy Donor | – | Female | 24 | Non-viral 6 factors: OSKM+LN | Simone et al. 2014 |
CTR AC 13 | Healthy Donor | – | Female | 24 | Non-viral 6 factors: OSKM+LN | Simone et al. 2014 |
