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Therapeutic RNA Targeting
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Summary
Therapeutic RNA Targeting
involves the use of
RNA molecules
or
RNA-targeting drugs
to modulate
Gene Expression
or correct
Genetic Disorders
, offering a precise approach to treat
Diseases at their molecular root
. This strategy leverages the
Natural roles of RNA
in
Gene Regulation
and
Protein Synthesis
, enabling the development of
Highly specific therapies
for a wide range of conditions, including cancers,
Viral Infections
, and
Genetic Diseases
.
Concepts
RNA Interference (RNAi)
Antisense oligonucleotides (ASOs)
CRISPR-Cas9 for RNA editing
Small Interfering RNA (siRNA)
MicroRNA (miRNA) therapeutics
RNA splicing modulation
RNA vaccines
Gene Silencing
RNA-based gene therapy
Targeted RNA degradation
Cas13
RNA targeting
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