Educational explorer

Different diseases.
A shared stop signal.

Explore KritRNA’s three initial research programmes by disease, gene and validation question.

Hematology · India-priority

β-Thalassemia

HBB

Selected HBB nonsense variants can interrupt β-globin production. KritRNA is defining candidate and assay requirements for selected contexts.

Research questions
  • Which premature stop and local sequence context?
  • Which amino acid should be restored?
  • How will β-globin protein and function be measured?
Open official gene record ↗
Neuromuscular

Duchenne muscular dystrophy

DMD

Selected DMD nonsense variants can interrupt dystrophin production. The large gene and disease-relevant assay context create distinct design and validation constraints.

Research questions
  • Which transcript and exon context?
  • How will full-length dystrophin be distinguished?
  • What disease-relevant cellular model is appropriate?
Open official gene record ↗
Oncology · tumour suppressor restoration

TP53-mutant cancer

TP53

Selected TP53 nonsense mutations can prevent production of full-length p53. KritRNA is defining how suppressor-tRNA candidates could restore protein expression and tumour-suppressor function in appropriate cancer models.

Research questions
  • Which TP53 nonsense variant and tumour context?
  • Which amino acid restoration best preserves p53 function?
  • How will transcriptional activity and cancer-cell response be measured?
Open official gene record ↗
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