AAV	Adeno-associated virus
AAVs	Adeno-associated viruses
BBB	Blood–brain barrier
CDNA	complementary DNA
CNS	Central nervous system
CRISPR	Clustered Regularly Interspaced Short Palindromic Repeat
CSF	Cerebrospinal fluid
DS	Dermatan sulphate
ERT	Enzyme replacement therapy
FDA	Food and Drug Administration
FGF	Fibroblast growth factor
GAGs	Glycosaminoglycans
GC	Genome copies
GvHD	Graft versus host disease
gRNA	guide RNA
HS	Heparan sulphate
HSCT	Hematopoietic stem cell transplantation
HR	Homologous recombination
iPSCs	induced pluripotent stem cells
KSPD	Kyoto Scale of Psychological Development 2001
LSDs	Lysosomal storage disorders
MPS I	Mucopolysaccharidosis type I
MPS II	Mucopolysaccharidosis type II
MPS IIIA	Mucopolysaccharidosis type IIIA
NGS	Next Generation Sequencing
NHEJ	Nonhomologous end joining
PAM	Protospacer adjacent motif
SCMAS	Subunit c of mitochondrial ATP synthetase
SNV	Single nucleotide variant
TALENs	Transcription activator-like effector nucleases
TLR4	Toll-like receptor 4
TNF-α	Tumour necrosis factor α
Vg	vector genomes
ZFNs	Zinc finger nucleases