Charlotte Rohde Knudsen

Developing Peptide Therapeutics for diseases caused by Nonsense Mutations: from Hit to Lead

Applicant

Charlotte Rohde Knudsen

Project description

Our genomic DNA codes for proteins that our cells require to function properly. Mutations may alter the protein code and cause the production of faulty proteins. Nonsense mutations represent a specific type of DNA mutations that turn an amino acid-encoding codon into a stop codon. This causes the ribosome to terminate translation of the genetic message prematurely, and results in the production of truncated, non-functional proteins. This condition leads to a variety of incurable diseases from muscular dystrophies to cancer.

Diseases caused by nonsense mutations can in theory be treated medically, but currently available chemicals suffer from severe toxicity or low efficiency. These drugs are therefore of limited clinical relevance, and novel strategies for the discovery of safe drugs are needed.

We have developed and applied a novel strategy to uncover nontoxic peptide-based drugs that can bypass disease-causing nonsense mutations. Our ultimate goal is to develop the identified drug candidates into a general cure of diseases caused by nonsense mutations for the benefit of millions of patients worldwide.

Institution

Aarhus University