Table 2.

Summary of the characteristics of the seven transgenic marmosets

IDSexCAG repeatsChromosomes with transgene integrationDisease onsetDisease progression
PQD1Male1201, 4, 174 monthsModerate
PQD2Female1201, 17, 223 monthsSevere
PQD3FemaleND
PQD4Female120
PQD5Female120, 80
PQD6Female1207, 224 monthsMild
PQD7Male120, 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.