An RNA therapy is designed to correct the mistake, or mutation, in the RNA of someone with a genetic disease. By correcting the mistake, the RNA can then be used to create the protein that the cell needs, taking away the underlying cause of the disease.
How RNA therapy works
How RNA therapy works
Why focus on RNA therapies?
RNA therapies can perform the same functions as other types of medicines but have broader applications. If a genetic disease results in a missing or broken protein, an RNA therapy can often correct this. Research at ProQR and other companies has shown promising results and other approved RNA therapies are helping people around the world.
What is RNA?
RNA stands for ribonucleic acid and it is an essential component of all living cells. RNA is used for ‘translation’, the process in which proteins are created in a cell. RNA itself is produced from DNA, in a process called ‘transcription’.
How is RNA therapy different from gene therapy and CRISPR?
Just like gene therapy and CRISPR approaches, RNA therapies are genetic therapies that aim to take away the underlying cause of a genetic disease. However, the fundamental approach is different. Gene therapy and CRISPR approaches make changes to DNA. These changes are permanent, with the risk of permanent side-effects. Unlike gene therapy, RNA therapies aim to repair RNA, which is a product of DNA, therefore any changes made to the RNA are reversible. RNA therapies are much smaller than gene therapies making manufacturing and delivery easier.
Targeted therapy and precision medicine
All RNA therapies are unique. Every molecule is designed to fit a specific mutation in the RNA with the aim to repair it, making it a highly precise and targeted approach. Scientists at ProQR design our drug candidates using the newest technologies available. We then test our RNA therapies in retinal organoid models, this is a state-of-the-art technique in which patient skin cells are used to grow a mini retina in the laboratory. This way we hope to predict which candidate performs best in humans.
ProQR’s RNA therapies are what scientists call antisense oligonucleotides, or AON or ASO treatment. They consist of short stretches of synthetic RNA that are chemically modified to increase efficiency and uptake into cells. This technique is an established approach to treat genetic diseases and there are approved RNA therapies being used to treat patients today.