Table 1.
Patient | Disease |
iPSC |
|||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Code | Sex | Agea | Age onset | Family history | Mutation | Initial symptomsb | LDopa response | # of Lines | Clones selected | Karyotype | Transgene silencingc | Pluripotency markersc | In vitro differentiationc | Teratoma assayc | |
CONTROL | SP09 | M | 66 | 4 | SP09.2 | 46,XY | Passed | Passed | Passed | N/P | |||||
SP09.4 | 46,XY | Passed | Passed | Passed | Passed | ||||||||||
SP11 | F | 48 | 3 | SP11.1 | 46,XX | Passed | Passed | Passed | Passed | ||||||
SP11.4 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
SP15 | F | 47 | 4 | SP15.2 | 46,XX | Passed | Passed | Passed | Passed | ||||||
SP15.3 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
SP15.4 | 47,XX + 20 | Passed | Passed | N/P | N/P | ||||||||||
SP17 | M | 52 | 3 | SP17.1 | 47,XY + 20 | Passed | Passed | Passed | N/P | ||||||
SP17.2 | 46,XY | Passed | Passed | Passed | Passed | ||||||||||
SP17.3 | 46,XY | Passed | Passed | Failed | N/P | ||||||||||
ID-PD | SP01 | F | 63 | 58 | No | No | T and B | N/A | 4 | SP01.1 | 46,XX | Passed | Passed | Passed | Passed |
SP01.4 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
SP02 | M | 55 | 48 | No | No | T | N/A | 2 | SP02.1 | 46,XY | Passed | Passed | Passed | Passed | |
SP02.2 | 46,XY | Passed | Passed | Passed | N/P | ||||||||||
SP04 | M | 46 | 40 | No | No | B | Good | 2 | SP04.1 | 46,XY | Passed | Passed | Passed | N/P | |
SP04.2 | 46,XY | Passed | Passed | Passed | Passed | ||||||||||
SP08 | F | 66 | 60 | No | No | T | Good | 4 | SP08.1 | 46,XX | Passed | Passed | Passed | Passed | |
SP08.2 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
SP08.3 | 46,XX | Failed | N/P | N/P | N/P | ||||||||||
SP10 | M | 58 | 50 | No | No | D | Good | 2 | SP10.1 | 46,XY | Passed | Passed | Passed | N/P | |
SP10.2 | 46,XY | Passed | Passed | Passed | Passed | ||||||||||
SP14 | M | 55 | 51 | No | No | B | Good | 2 | SP14.1 | 46,XY | Passed | Passed | Passed | Passed | |
SP14.2 | 46,XY | Passed | Passed | Passed | N/P | ||||||||||
SP16 | F | 51 | 48 | No | No | B | N/A | 4 | SP16.2 | 46,XX | Passed | Passed | Passed | Passed | |
SP16.3 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
LRRK2-PD | SP05 | M | 66 | 52 | Yes | LRRK2 | B | Good | 2 | SP05.1 | 46,XY | Passed | Passed | Passed | Passed |
SP05.2 | 46,XY | Passed | Passed | Passed | N/P | ||||||||||
SP06 | M | 44 | 33 | Yes | LRRK2 | T | Good | 6 | SP06.1 | 46,XY | Passed | Passed | N/P | N/P | |
SP06.2 | 46,XY | Passed | Passed | Passed | Passed | ||||||||||
SP12 | F | 63 | 49 | Yes | LRRK2 | T | Good | 4 | SP12.3 | 46,XX | Passed | Passed | Passed | Passed | |
SP12.4 | 46,XX | Passed | Passed | Passed | N/P | ||||||||||
SP13 | F | 68 | 57 | Yes | LRRK2 | T | Good | 4 | SP13.2 | 46,XX | Passed | Passed | Passed | N/P | |
SP13.4 | 46,XX | Passed | Passed | Passed | Passed |
N/A, information not available; N/P, test not performed.
Age at biopsy.
T, tremor; B, bradykinesia; D, foot dystonia.
Tests performed as exemplified in Fig 1.