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

Table 5 Body weight

Peak BW untreated Npc-/-	Peak BW treated Npc-/-	Vector	Administration	References
n.a.*				[16]
6 weeks	14 weeks	AAV-PHP	Systemic	[11]
6 weeks	8 weeks	AAV9		[11]
7 weeks	15 weeks	AAV9/3	CNS	[12]
6 weeks	12 weeks	AAV9 low dose	CNS	[15]
6 weeks	16 weeks	AAV9 high dose	CNS	[15]
8 weeks	18 weeks (f)	AAVrh.10	CNS	[14]
8 weeks	16 weeks (m)	AAVrh.10	CNS	[14]
n.a.				[13]
6 weeks	8 weeks	AAV9-CamKII	Systemic	[10]
6 weeks	12 weeks	AAV9-EF1α	Systemic	[10]
6 weeks	6 weeks	TfRMAb targeted THLs	Systemic	[22]

BW bodyweight, f females, m males, n.a. not assessed, *peak body weight is not assessed, CNS central nervous system

ISSN: 1750-1172