AADC	Aromatic l-amino acid decarboxylase
AAV	Adeno-associated virus
ADA-SCID	Adenosine deaminase-deficient severe combined immunodeficiency
AFM	Atomic force microscopy
ARSA	Deficiency of lysosomal arylsulfatase A
ASOs	Antisense oligonucleotides
cAMP	Cyclic adenosine monophosphate
CD	Circular dichroism
cDNA	Complementary deoxyribonucleic acid
CF	Cystic fibrosis
CFTR	Cystic fibrosis transmembrane conductance regulator
CLSM	Confocal laser scanning microscopy
CMV	Cytomegalovirus
CNS	Central nervous system
CPP	Cell penetrating peptide
CRISPR	Clustered regularly interspaced short palindromic repeats
CS	Chitosan
DA	Degree of acetylation
DHM	Digital holographic microscopy
DLS	Dynamic light scattering
DP	Degree of polymerization
DS	Dextran sulphate
dsRNA	Double-stranded ribonucleic acid
eGFP	Green fluorescent protein
ENaC	Epithelial sodium channel
GAN	Giant axonal neuropathy
HA	Hyaluronic acid
HDP	High degree of polymerization
HSC	Hematopoietic stem cells
ITC	Isothermal titration calorimetry
LDV	Laser Doppler velocimetry
LNA	Locked nucleic acids
LPLD	Familial lipoprotein lipase deficiency
LSD	Lysosomal storage diseases
miRNA	Micro ribonucleic acid
MLD	Methachromatic leukodistrophy
mRNA	Messenger ribonucleic acid
Mw	Molar mass
N/P	Amino to phosphate charge ratio
NPs	Nanoparticles
oc pDNA	Open chain plasmid deoxyribonucleic acid
PA	Pattern of acetylation
PAMPs	Pathogen-associated molecular patterns
PCD	Primary ciliary dyskinesia
PDE	Phosphodiesterase
pDNA	Plasmid deoxyribonucleic acid
PEI	Polyethylene imine
PGA	Poly-d-glutamic acid
PLL	Poly-Lysine
rAAV	Recombinant adeno-associated virus
rAD	Recombinant adenovirus
RGMC	Reduced generation of multiple motile cilia
RISC	RNA interference silencing complex
rLV	Recombinant lentivirus
RT-PCR	Real-time quantitative reverse transcription polymerase chain reaction
SAXS	Small angle X-ray scattering
SCID	Severe combined immunodeficiency
SeV	Murine parainfluenza virus type 1
siRNA	Small interfering ribonucleic acid
sc pDNA	Supercoiled plasmid deoxyribonucleic acid
SPR	Surface plasmon resonance
ssRNA	Single-stranded ribonucleic acid
TALEN	Transcription activator-like effector nucleases
TEM	Transmission electron microscopy
TLR	Toll-like receptor
TPP	Pentasodium tripolyphosphate
WAS	Wiskott-Aldrich syndrome
ZFN	Zinc finger nucleases