RNA therapeutics

E621880

RNA therapeutics are medical treatments that use RNA molecules to modulate gene expression or protein production for preventing, treating, or curing disease.

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Statements (52)

Predicate Object
instanceOf biomedical technology
molecular medicine approach
therapeutic modality
aimsTo cure disease
prevent disease
treat disease
commonlyUses conjugate delivery systems
lipid nanoparticles
polymeric nanoparticles
enables personalized medicine
rapid vaccine development
facesChallenge RNA instability
immune activation
off-target effects
targeted delivery
includesSubtype RNA aptamer therapeutics
RNA vaccine
antisense oligonucleotide therapeutics
guide RNA-based genome editing therapeutics
mRNA therapeutics
miRNA therapeutics
self-amplifying RNA therapeutics
siRNA therapeutics
isFieldOf drug development
translational research
mechanismOfAction gene silencing
gene upregulation
protein replacement
receptor binding
splicing modulation
translation inhibition
regulatoryApprovedExample COVID-19 mRNA vaccines
eteplirsen NERFINISHED
givosiran NERFINISHED
inclisiran NERFINISHED
inotersen NERFINISHED
mipomersen NERFINISHED
nusinersen NERFINISHED
patisiran NERFINISHED
requires delivery system
targetsProcess gene expression
protein production
treats cancer
genetic diseases
infectious diseases
metabolic disorders
neurological disorders
usesMoleculeType RNA
usesStrategy chemical modification of nucleotides
codon optimization
formulation optimization
sequence optimization

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Full triples — surface form annotated when it differs from this entity's canonical label.

Drew Weissman hasResearchInterest RNA therapeutics