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. 2025 Dec 12;13(12):3066. doi: 10.3390/biomedicines13123066
SCA1 Spinocerebellar ataxia type 1
CAG cytosine–adenine–guanine
ATXN1 ataxin-1
Ser776 serine 776
ASO antisense oligonucleotide
RNAi RNA interference
NfL neurofilament light protein
iPSC induced pluripotent stem cell
NLS nuclear localization signal
UPS ubiquitin–proteasome system
ATXN1-CIC ataxin-1-Capicua
CIC Capicua
HMGB ½ High-Mobility Group Box ½
LANP leucine-rich acidic nuclear protein
PKA protein kinase
RBM17 RNA-Binding Motif Protein 17
U2AF65 U2 snRNP auxiliary factor 65 kDa
NLK Nemo-like kinase
ISG15 Interferon-stimulated gene 15
GFI1 growth factor independence 1
SMRT Silencing Mediator of Retinoid and Thyroid Receptors
Tip60 Tat-interacting protein 60
RORα retinoid-related orphan receptor α
Boat1 ATXN1-like paralog
RPA replication protein A
ASR acoustic startle response
FST forced swim test
CaB calbindin
PV Parvalbumin
CCK cholecystokinin
LPS lipopolysaccharide
Ara-C Cytarabine