mRNA Therapy for Cystic Fibrosis

mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.

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In this article
3D illustration of mRNA and ribosomes inside a cell
  • mRNA therapy would deliver a new, correct copy of CFTR mRNA to cells, allowing them to produce healthy CFTR protein.
  • This treatment would not affect a person’s DNA or CFTR mutations.
  • mRNA therapy is not permanent and would need to be re-dosed regularly to be effective.

What Is mRNA Therapy?

Genes are specific sequences of DNA that carry the instructions for making proteins. The cystic fibrosis transmembrane conductance regulator (CFTR) gene provides cells with the instructions to make CFTR protein. However, when making a protein, the cell does not use the DNA directly. Instead, the cell copies the genetic instructions into a similar molecule called messenger RNA (mRNA). The mRNA then acts as a template, carrying the instructions from the gene to the protein-making machinery in the cell. If our genes were like pages in an instruction manual, mRNA would be photocopies of the pages. These copies are taken to the cellular construction site where the protein can be assembled.

There are several different ways to provide the correct genetic instructions to cells. Gene therapy involves providing a new, nonmutated copy of the CFTR gene, or DNA, so that cells can make their own mRNA copies. Another approach is just to give cells the mRNA copies directly. mRNA therapy would deliver a new, correct copy of CFTR mRNA to cells, allowing them to produce healthy CFTR protein. This type of therapy could potentially work for any person with CF, regardless of their CFTR mutations.

Watch this video to see how this process might work.

Advantages and Challenges of mRNA Therapy

An advantage of mRNA therapy is that it does not affect a person’s DNA or CFTR mutations. Unlike gene editing or integrating gene therapy, there is no risk of disrupting a person's genome.

A disadvantage is that mRNAs are naturally broken down quickly inside cells, so the effects of the mRNA therapy might last only for a short time, such as one or two weeks. This would mean that the treatment would likely need to be re-dosed regularly for it to continue to work.

Watch this video to learn how different genetic therapies, including mRNA therapy, gene therapy, and gene editing, could restore CFTR function in lung cells.

mRNA therapies are being developed to treat a wide range of diseases, and several potential mRNA therapies for CF are currently in our Drug Development Pipeline.

To hear more about genetic therapies in the pipeline and how they might work, watch this webinar featuring CF researchers Deepika Polineni, MD, MPH, and George M. Solomon, MD.

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