Curriculum
Module 10 · 65 min
RNA Biology & Translation
Splicing, ncRNAs, mRNA capping and the ribosome — and the drugs that reach them.
CoreClinicalResearch
Topics
What this module covers
- 01Pre-mRNA splicing, alternative splicing, splicing-modulator therapies (nusinersen, risdiplam)
- 025' cap, 3' polyadenylation, RNA stability
- 03Ribosome structure and translation cycle
- 04Non-coding RNAs: miRNA, lncRNA, circRNA, piRNA
- 05RNA modifications (m6A) and the epitranscriptome
- 06mRNA vaccines and lipid nanoparticle delivery
Deep dives
Lesson sub-pages
Learning objectives
By the end of this module you will be able to
- L01Explain how nusinersen and risdiplam restore SMN2 exon-7 inclusion in spinal muscular atrophy.
- L02Compare aminoglycoside vs macrolide mechanism on the bacterial ribosome.
- L03Outline the design choices in an mRNA vaccine: nucleoside modification, cap, LNP composition.
Expected takeaways
What you should walk away believing
- →Splicing is regulated, druggable, and frequently disrupted in disease.
- →mRNA technology is now a platform — vaccines, protein replacement, and editing payload delivery.
- →Antisense oligonucleotides (ASOs) and siRNAs are an established therapeutic modality.
Core summary
At the Core level
Genes are not directly read into protein; they are transcribed into pre-mRNA, spliced, capped, polyadenylated, exported, translated, and quality-controlled. Each of these steps is regulated and can fail in disease. Modern therapeutics now intervene at each step — splice modulators, ASOs, siRNAs, mRNA vaccines, and small molecules that bind RNA.
Evidence-graded claims
Claims, scored A–F
A
mRNA vaccines work by translation of antigen mRNA in transfected cells
Mechanism confirmed; pseudouridine-modified mRNA in LNPs.
A
siRNA therapeutics can silence specific transcripts in liver
GalNAc-conjugated siRNAs (inclisiran, patisiran) are clinically used.
D
All circulating cell-free miRNAs are reliable biomarkers
Variable preanalytics; specific contexts work, broad use is overstated.
Quiz
Check your understanding
Q1. Which therapy increases SMN2 exon-7 inclusion?
Q2. Why is pseudouridine used in mRNA vaccines?
Flashcards
Lock it in
1 / 3
Front
Two SMA therapies and modality?
Click to flip
Suggested reading
Primary literature
- mRNA vaccines — a new era in vaccinology — Pardi et al., Nat Rev Drug Discov 2018 ↗
- Splicing modulators in spinal muscular atrophy — Mercuri et al., Lancet Neurol 2022 ↗