AAV	adeno-associated virus
AGO2	argonaute 2
AIDS	acquired immunodeficiency syndrome
ALS	amyotrophic lateral sclerosis
ASO	antisense oligonucleotide
ATXN2	ataxin-2
BBB	blood–brain barrier
Cas	CRISP-associated
CNS	central nervous system
CPP	cell-penetrating peptide
CSF	cerebrospinal fluid
C9orf72	chromosome 9 open reading frame 72
DM	myotonic dystrophy
DMD	Duchenne muscular dystrophy
DMPK	dystrophia myotonic protein kinase
DNA	deoxyribonucleic acid
dsRNA	double-stranded RNA
EAP	expanded access program
fALS	familial ALS
FDA	Food and Drug Administration
FKRP	fukutin-related protein
FLTD-TDP	frontotemporal lobar degeneration with TDP-43 inclusions
FTD	frontotemporal dementia
FUS	fused in sarcoma
GRT	gene-replacement therapy
HGF	hepatocyte growth factor
ILI	isolated limb infusion
IM	intramuscular
ISS-N1	intronic splicing silencer-N1
IT	intrathecal
IV	intravenous
LGMD	limb-girdle muscular dystrophy
mRNA	messenger RNA
miRNA	microRNA
OLE	open-label extension
PMO	phosphorodiamidate morpholino oligomer
polyQ	polyglutamine
PS	phosphorothioate
RISC	RNA-induced silencing complex
RNA	ribonucleic acid
RNAi	RNA interference
SMA	spinal muscular atrophy
SMN	survival of motor neuron
siRNA	small interfering RNA
SOD1	superoxide dismutase 1
TARDBP	TAR DNA-binding protein
TDP-43	transactive response DNA-binding protein 43
tMCK	muscle-specific creatine kinase (promoter)
UTR	untranslated region
vg	viral genome
2′-O-Me	2′-O-Methyl
2′-O-MOE	2′-O-methoxy ethyl/phosphorothioate