AAP	Assembly-Activating Protein
AAV	Adeno-Associated virus
ALS	Amyotrophic Lateral Sclerosis
ASO	Antisense oligonucleotides
B6.SOD1-G93A	SOD1-G93A mouse model backcrossed the to the C57BL/6J
BAC	Bacterial artificial chromosome
BDNF	Brain derived neurotrophic factor
C9-100-1000	BAC C9orf72 mice harboring from 100 to 1000 repeats
C9-450	BAC C9orf72 mice harboring 450 repeats
C9-500	BAC C9orf72 mice harboring 500 repeats
C9orf72	Chromosome 9 open reading frame 72
CHMP	Committee for Medicinal Products for Human Use
CNS	Central nervous system
CRISPR	Clustered regularly interspaced short palindromic repeats
CSF	Cerebrospinal fluid
DPRs	Dipeptide repeat proteins
EIAV	Equine infectious anaemia virus
EMA	European Medicines Agency
fALS	Familial ALS
FDA	Food and Drug Administration
FTD	Frontotemporal dementia
FUS	Fused in sarcoma
G4C2	GGGGCC
G93A	single amino acid substitution of glycine to alanine at codon 93 of the human SOD1
GA	Glycine–alanine
GDNF	Glial cell line derived neurotrophic factor
GP	Glycine–proline
GR	Glycine–arginine
GWAS	Genome-wide association studies
HiRet	Highly-efficient retrograde gene transfer
HIV	Human immunodeficiency virus
HRE	Hexanucleotide repeat expansion
IGF-1	Insulin-like growth factor 1
iPSC	Induced pluripotent stem cells
ITRs	Inverted terminal repeats
IV	Intravenous
LTR	Long terminal repeats
LV	Lentiviral vectors
miR	microRNA
MN	Motor neurons
MND	Motor neuron disease
MNDs	Motor neuron disorders
NeuRet	Neuron-specific retrograde gene transfer
NMD	Nonsense Mediated Decay
PA	Proline–alanine
PR	Proline–arginine
RAN	Repeat-associated non-AUG-dependent translation
RBPs	RNA-binding proteins
RISC	RNA-induced silencing complex
RNAi	RNA interference
RNase H	Ribonuclease H
RVG	Rabies virus envelope glycoprotein
sALS	Sporadic ALS
Sc	Self-complementary
shRNA	Short hairpin RNA
siRNA	Small interfering RNA
SMA	Spinal Muscular Atrophy
SMN	Survival of motor neuron
SOD1	Cu/Zn superoxide dismutase 1 enzyme
SOD1-G93A	SOD1 mouse model in the mixed B6SJL genetic backgroundexpressing multiple copies of the human SOD1 transgene, carrying a single amino acid substitution of glycine to alanine at codon 93
Ss	Single-stranded
TDP-43	TAR DNA binding protein of 43 kDa
VEGF	Vascular endothelial growth factor
VSV-G	Vesicular stomatitis virus glycoprotein