AADC	Aromatic L-amino acid decarboxylase
AAV	Adeno-Associated Virus
ABE	Adenine Base Editor
AD	Alzheimer’s Disease
AdV	Adenoviral Vector
AHC	Alternating Hemiplegia of Childhood
AI	Artificial Intelligence
ALS	Amyotrophic Lateral Sclerosis
ASD	Autism Spectrum Disorder
ASO	Antisense Oligonucleotide
BBB	Blood–Brain Barrier
BD	Bipolar Disorder
BDNF	Brain-Derived Neurotrophic Factor
BE	Base Editing
BLA	Biologics License Application
CAGR	Compound Annual Growth Rate
CED	Convection-Enhanced Delivery
CGT	Cell and Gene Therapy
CMV	Cytomegalovirus
CNS	Central Nervous System
CRISPR	Clustered Regularly Interspaced Short Palindromic Repeats
CRISPRa	CRISPR activation
CRISPRi	CRISPR interference
DALY	Disability-Adjusted Life Year
dCas9	Catalytically inactive “dead” Cas9
DNA	Deoxyribonucleic acid
DSB	Double-Strand Break
ECM	Extracellular Matrix
EV	Extracellular Vesicle
FDA	U.S. Food and Drug Administration
FUS	Focused Ultrasound
GDNF	Glial Cell Line-Derived Neurotrophic Factor
GMP	Good Manufacturing Practice
HD	Huntington’s Disease
HDR	Homology-Directed Repair
HTT	Huntingtin
Indel	Insertion or deletion
iPSC	Induced Pluripotent Stem Cell
kb	Kilobase
LNP	Lipid Nanoparticle
MCI	Mild Cognitive Impairment
MENP	Magneto-electric Nanoparticle
miRNA	MicroRNA
mRNA	Messenger RNA
MSC	Mesenchymal Stem Cell
NAM	New Approach Methodology
NBS	Newborn Screening
NfL	Neurofilament light chain
NHEJ	Non-Homologous End Joining
PAM	Protospacer Adjacent Motif
PD	Parkinson’s Disease
PE	Prime Editing
pegRNA	Prime editing guide RNA
PK/PD	Pharmacokinetic and pharmacodynamic
PNP	Polymeric Nanoparticle
RIDE	Ribonucleoprotein Delivery system
RISC	RNA-induced silencing complex
RNA	Ribonucleic acid
RNP	Ribonucleoprotein
RT	Reverse Transcriptase
RVD	Repeat-variable diresidue
sgRNA	Single-guide RNA
siRNA	Small interfering RNA
SMA	Spinal Muscular Atrophy
TALEN	Transcription Activator-Like Effector Nuclease