ZFN	Zinc-finger Nucleases
TALEN	Transcription Activator-like Effector Nucleases
CRISPR	Clustered Regularly Interspaced Short Palindrome Repeats
hiPSCs	Human induced Pluripotent Stem Cells
hPSCs	Human Pluripotent stem cells
hESCs	Human Embryonic Stem Cells
HIV-1	Human Immunodeficiency Virus
tracrRNA	trans-activating crRNA
sgRNA	single-guide RNA
IV	Intravenous
saCas9	Staphylococcus aureus Cas9
MPS II	Mucopolysaccharidosis II
IDS	Iduronate 2-Sulfatase
CNS	Central Nervous System
HPV	Human Papillomavirus
HTI	Hereditary Tyrosinemia Type I
AMD	Age-related Macular Degeneration
DMD	Duchenne Muscular Dystrophy
ALS	Amyotrophic Lateral Sclerosis
OTC	Ornithine Transcarbamylase
AAT	Alpha-1 antitrypsin
LCA	Leber’s congenital amaurosis
HR	Homologous Recombination
DTA	Diphtheria A
CCR	Chemokine Co-Receptor
ERT	Enzyme Replacement Therapy
ART	Anti-retroviral therapy
NSG	NOD/SCID/IL2rγnull
SOD1	Superoxide Dismutase 1
rAAV	Recombinant Adeno-Associated Viruses
AAV	Adeno-Associated Viruses
BLT	Bone marrow/liver/thymus
SMA	Spinal Muscular Atrophy
MHC	Major Histocompatibility Complex
PLNP	Polyethylene glycol phospholipid-modified cationic lipid nanoparticle
CNS	Central nervous system
BBB	Blood brain barrier
MOI	Multiplicity of infection
NPs	Nanoparticles