Table 8.

Summarizes the suitability of other polyQ diseases for the evaluation of experimental therapies

	SCA1 B05	SCA1 154Q/2Q	SCA2 58Q	SCA3 polyQ69	SCA3 MJD84.2	SCA3 70.61 CAG	SCA3 Q79	SCA7 90Q R7E	SCA17 L7-hTBP	SBMA AR-97Q	SBMA 112Q	DRPLA Atro 118Q
Construct validity—genetic similarity to the human patients (full length protein/natural promoter/targeted transgene integration)	+h/−/−	+hb/+/+	+h/−/−	−/−/−	+h/+/−	+h/−/−	+h/−/−	+h/−/−	+/−/−	+h/−/−	+/−/−	+h/−/−
Face validity—phenotypic similarity to the human patients (specific cell loss/rotarod impairment/ cognitive alterations)	+/+/+	+/+/+	+/+/−	−/+/−	+/+/−	−/+/−	−/+/−	−/−/− (ret)	+/+/−	−/+/−	+/+/+	−/+/−
Number of therapeutic approaches published	6	8	2	1	1	1	1	2	1	11	1	1
Number of phenotypes identified	18	27	10	12	36	18	17	14	24	25	23	19
Phenotype progression (AD50; age at 50 % detected phenotypes)	10	19.5	25	3	41	13	30	3.25	13	10.5	30	11.5
Breeding and husbandry (severe phenotype/reduced fertility)	+/nr	+/nr	nr	+/nr	nr	+/nr	nr	nr	+/nr	+/nr	−/+	+/nr

This suitability can be evaluated by using construct validity (genetic similarities), face validity (phenotypic similarities), and predictive validity (cannot be determined at present). Moreover, this suitability can be assessed by the number of therapeutic approaches published (based on the data table) and the number of phenotypes identified (based on the data table in part I of the review). Additionally, the AD50 parameter (expressed as the number of weeks) reflects the disease dynamic in the models (see review part I for detailed information). The separate issue in assessing the therapeutic suitability in mouse models is the model maintenance and breeding

h human sequence, hb hybrid human/mouse sequence, nr not reported, ret 90Q R7E phenotype is limited to the eye retina