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

Table 6 Survival

Median survival untreated Npc-/-	Median survival treated Npc-/-	Lifespan extension (%)	Vector	Longest survival	References
76 days	263 days	246	AAV-NPC1	275 days	[16]
72 days	234 days	225	AAV-PHP	> 1 year (AAV-PHP)	[11]
72 days	112 days	56	AAV9		[11]
75 days	205 days	173	AAV9/3	310 days	[12]
75 days	116.5 days	55	AAV9 low dose	126 days	[15]
75 days	158 days	111	AAV9 high dose	168 days	[15]
112 days (f)	280 days (f)	150	AAVrh.10	280 days (f)	[14]
98 days (m)	168 days (m)	71	AAVrh.10	280 days (f)	[14]
71 days	94 days	32	AAV9	100 days	[13]
69 days	103 days (j)	49	AAV9-CamKII	140 days (j)	[10]
69 days	166 days	141	AAV9-EF1α	300 days	[10]
75 days*	75 days	n.s.	TfRMAb targeted THLs	77 days	[22]

f = females, m = males (euthanized due to the development of penile prolapse, starting at 24 weeks of age), j = juvenile at the time of injection, * = data are reported as mean, n.s. = non-significant

ISSN: 1750-1172