Table 2.

Reported sequestration of RNA-binding proteins into sense RNA foci in patient cells and tissue

RNA-binding protein	C9ALS or FTD	Patient-derived cells	Patient tissue	Study
ADARB2	ALS	iPSC-differentiated neurons	Motor cortex	[8▪▪]
ALYREF	ALS	ND	Cerebellum and spinal cord	[45▪]
hnRNP A1	ALS and ALS/FTD	iPSC-differentiated motor neurons		[22▪]
	ALS	ND	Cerebellum	[45▪]
hnRNP Ha	ALS	ND	Cerebellum	[24▪]
	ALS	Cerebellum and cerebellum and spinal cord		[45▪]
Nucleolin	ALS	ND	Motor cortex	[25▪]
Pur-alpha	ALS and ALS/FTD	iPSC-differentiated motor neurons		[22▪]
SRSF2 (SC35)	ALS	ND	Cerebellum	[24▪]
	ALS	ND	Cerebellum and spinal cord	[45▪]

ADARB2, adenosine deaminase, RNA-specific, B2; ALS, amyotrophic lateral sclerosis; ALYREF, Aly/REF export factor; FTD, frontotemporal dementia; hnRNP, heterogeneous nuclear ribonucleoprotein; iPSC, induced pluripotent stem cell; ND, not determined; SRSF2 (SC35), serine/arginine-rich splicing factor 2 (also known as SC35).

^aSequestration of hnRNP H into RNA foci was not observed in C9FTD iPSC-neurons [6^▪].