ID | Sex | CAG repeats | Chromosomes with transgene integration | Disease onset | Disease progression |
---|---|---|---|---|---|
PQD1 | Male | 120 | 1, 4, 17 | 4 months | Moderate |
PQD2 | Female | 120 | 1, 17, 22 | 3 months | Severe |
PQD3 | Female | ND | |||
PQD4 | Female | 120 | |||
PQD5 | Female | 120, 80 | |||
PQD6 | Female | 120 | 7, 22 | 4 months | Mild |
PQD7 | Male | 120, 43 |
ND, not determined.
Although all marmosets except PQD3 carried the ataxin 3-120Q transgene, transgenes with other CAG repeat lengths were also found to be expressed in PQD5 and 7, respectively. PQD1, 2, and 6 had several integration sites and cellular mosaicism and developed neurologic symptoms of varying degrees at 3–4 months after birth.