Complement is dispensable for neurodegeneration in Niemann-Pick disease type C

Table 1 Deficiency in other immune response-relevant pathways does not alter neurodegeneration in NPC disease

*Npc1* ^-/- *& ____*	Function/Pathway	Mice^a(n)	Change in PN loss	Change in microglia	Change in %CD68^b	Ref.
C1qa ^-/-	Complement	11	No	No	P = 0.49	Figure 5B
C3 ^-/-	Complement	7	No	No	P = 0.88
Mmp12 ^-/-	ECM degradation	4	No	No	P = 0.95
Bcl2l11(Bim) ^-/-	Apoptosis	4	No	No	P = 0.17
Tlr7 ^-/-	Toll-like receptor	3	No	No	n/a
Unc93b1 ^3d/3d	Toll-like receptor	3	No	No	n/a
Pcp2-tTA; TetO-Npc1YFP	Purkinje neuron (PN) rescue	32	Yes	Yes	P = 0.0057	[2, 17]

^aNumber of mice used to observe PN loss and microglial activity for ages >P60, see Figure 5A and 5D.
^bTwo-way ANOVA: Does the gene affect %CD68 in the cerebellum or thalamus of Npc1 ^-/- mice compared to Npc1 ^-/- controls? P value <0.05 is considered significant; n = 4 for all mice at age P60. n/a means a statistical analysis was not performed for this group. Hippocampus and midbrain values, which showed no difference across samples, were used to normalize %CD68 in thalamus and cerebellum, respectively. Note: PN rescue corrects cerebellar but not thalamic pathology [17], P = 0.0005.

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