ABC-DLBCL

activated B-cell subtype of diffuse large B-cell lymphoma

ACE-I

angiotensin converting enzyme inhibitor

AGXT

alanine-glyoxylate aminotransferase

AIDS

acquired immunodeficiency syndrome

ALAS1

-aminolevulinic acid synthase

ALS

amyotrophic lateral sclerosis

AMD

age-related macular degeneration

ANGPTL3

angiopoietin-like 3

AOC

antibody-oligonucleotide conjugate

APC

antigen-presenting cell

apo (a)

apolipoprotein (a)

apoC-III

apolipoprotein C-III

ARB

angiotensin receptor blocker

ASO

antisense oligonucleotides

AT1

angiotensin 1 receptor

ATC

anaplastic thyroid cancer

CAD

coronary artery disease

CNS

central nervous system

CRISPR-Cas

clustered regularly interspaced short palindromic repeats-CRISPR-associated

CTCL

cutaneous T-cell lymphoma

CVD

cardiovascular disease

CXCL12

CXC chemokine ligand 12

DMD

Duchenne muscular dystrophy

EMA

European Medicines Agency

ESA

erythropoiesis-stimulating agents

FCS

familial chylomicronemia syndrome

FDA

Food and Drug Administration

GalNAc

N-acetylgalactosamine

HAO1

hydroxy acid oxidase 1

hATTR

hereditary transthyretin amyloidosis

HBsAg

hepatitis B surface antigen

HeFH

heterozygous familial hypercholesterolemia

HNRNPL

heterogeneous nuclear ribonucleoprotein L

HPV16

human papillomavirus 16

HSD17B13

hydroxysteroid 17-dehydrogenase 13

IE2

immediate early region 2

IGFIIR

insulin-like growth factor 2 receptor

iNeST

individualized neoantigen specific therapy

ISS-1

intronic splice silencing site-1

LCA10

Leber congenital amaurosis type 10

LDHA

lactate dehydrogenase A

LDL

low density lipoprotein

LICA

ligand-conjugated antisense

LNP

lipid-based nanoparticle

LR-MDS

lower-risk myelodysplastic syndromes

LRTD

lower respiratory tract disease

MASH

metabolic dysfunction-associated steatohepatitis

MCNS

minimal change nephrotic syndrome

MDD

major depressive disorder

MHC I

major histocompatibility complex class I

MHC II

major histocompatibility complex class II

MHRA

medicines and healthcare products regulatory agency

NAFLD

nonalcoholic fatty liver disease

NASH

nonalcoholic steatohepatitis

NSCLC

non-small cell lung cancer

PCSK9

proprotein convertase subtilisin/kexin type 9

PDCD4

programmed cell death 4

PDUFA

Prescription Drug User Fee Act

PH1

primary hyperoxaluria type 1

PNP

polymer-based nanoparticles

pre-RISC

premature RNAi-induced silencing complex

PTSD

post-traumatic stress disorder

RSV

respiratory syncytial virus

SERT

serotonin transporter

siRNA

short interfering RNAs

SMA

spinal muscular atrophy

SMN1

survival motor neuron 1

SMN2

survival motor neuron 2

SOD1

superoxide dismutase 1

SSO

splice-switching oligonucleotide

SSRI

selective serotonin reuptake inhibitors

T1D

type 1 diabetes patients

TPTE

trans-membrane phosphatase with tensin homology

VEGF

vascular endothelial growth factor

vLDL

very low-density lipoprotein

wtHTT

wild-type huntingtin