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. 2026 Feb 18;27(4):1953. doi: 10.3390/ijms27041953
CAIR
ATIC
5-aminoimidazole-4-carboxylate ribonucleotide
5-Aminoimidazole-4-carboxamide ribonucleotide formyltransferase
PPAT Amidophosphoribosyltransferase
ADA Adenosine Deaminase
ADK Adenosine Kinase
ADSL Adenylosuccinate Lyase
ALS Amyotrophic Lateral Sclerosis
C9orf72
CHX
Chromosome 9, Open Reading Frame 72
Cycloheximide
DNPB De novo purine biosynthesis
DPR Dipeptide Repeat Protein
CD73 Ecto-5′-nucleotidase
EV
EAAT2
Empty Vector
Excitatory amino acid transporter 2
FGAMS Formylglycinamide ribonucleotide amidotransferase
poly-GA Glycine-Alanine
poly-GR Glycine-Arginine
poly-GP Glycine-Proline
HRE Hexanucleotide Repeat Expansion
HGPRT Hypoxanthine-Guanine Phosphoribosyltransferase
iNPC Induced Neural Progenitor Cell
iAstrocyte Induced Neural Progenitor Cell-Derived Astrocyte
IMP Inosine Monophosphate
LLPS
LV-ADA
LV-EV
NV
Liquid−Liquid Phase Separation
lentivirus expressing ADA
lentivirus expressing empty vector
non-virally treated
PAICS Phosphoribosylaminoimidazole carboxylase/phosphoribosylaminoimidazole succinocarboxamide synthetase
GART Phosphoribosylglycinamide formyltransferase/phosphoribosylglycinamide synthetase/phosphoribosylaminoimidazole synthetase
poly-PA Proline-Alanine
poly-PR Proline-Arginine
PNP Purine Nucleoside Phosphorylase
SCID
SRSF1
SAICAR
RuvBL2
Severe Combined Immunodeficiency
Serine/arginine-rich splicing factor 1
succinylaminoimidazole carboxamide ribonucleotide
RuvB-like 2
XO Xanthine Oxidase