The Problem
Premature termination codons affect millions globally, yet therapeutic options remain limited
What Are Premature Termination Codons?
"A single wrong 'period' in the genetic sentence can silence an entire protein story."
Premature termination codons (PTCs) act like stop signs placed too early in a genetic message. When ribosomes—the cellular machines that read RNA—encounter these incorrect stops, they halt protein production mid-sequence.
The result: truncated, non-functional proteins that cause devastating rare diseases affecting millions of patients worldwide.
Understanding Translation
Normal Translation
Full-length protein
mRNA is read continuously by the ribosome, producing a complete, functional protein.
Premature Stop (PTC)
Truncated protein
A nonsense mutation creates an early stop codon, resulting in a shortened, non-functional protein.
Suppressor tRNA Concept
Rescuing translation
Engineered suppressor tRNAs read through the premature stop, restoring protein function.