Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines

Table 2 Study design

References	Sample size	Group size			Randomization/ Blinding	Study period*	Statistics^**
References	Sample size	WT	CTRL	T	Randomization/ Blinding	Study period*	Statistics^**
[16]	n.s. [66]	6	6	54	−/−	10 weeks	−
[11]	55	13	13	29	+ / +	9 weeks	+
[12]	n.s. [46]	11	23	12	−/−	11 weeks	−
[22]	36	n.s.	18	18	−/−	5 weeks	N
[15]	n.s. [38]	6	8	24	−/−	Up to 17 weeks^#	−
[14]	n.s.	n.s.	n.s.	n.s.	−/−	10 weeks	N
[13]	n.s.	n.s.	n.s.	n.s.	−/−	8 weeks	−
[10]	n.s. [44]	0	22	22	−/ +	9 weeks	−

WT wild-type, CTRL untreated Npc-/- mice, T treated Npc-/- mice, *refer to the duration of the study used for immunohistochemical evaluation (from injection to euthanasia), **fulfillment of the criteria stated in the ARRIVE guidelines, n.s. = not specified, e.g. the number of animals is not stated clearly in the method sections or elsewhere, N = statistical section is lacking, [calculated], #untreated Npc-/- were euthanized at end-stage of the disease at 9 weeks of age

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