CF Trust, CF Foundation Launch Gene-editing Project

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by Marisa Wexler MS |

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The U.K. Cystic Fibrosis Trust, in collaboration with the U.S. Cystic Fibrosis Foundation, is launching a new strategic research center (SRC) dedicated to studying gene editing as a potential treatment strategy for cystic fibrosis (CF).

“Making gene editing a reality for people with CF will take the coming together of expertise from across the world. That’s why we’re excited about being able to work in partnership with the CF Foundation to fund our newest, collaborative SRC,” Lucy Allen, PhD, director of research at the CF Trust, said in a press release.

“Advancing gene editing therapies is a top priority for the CF Foundation, but it will require collaboration to address challenging basic questions. By working with the UK CF Trust to support this Strategic Research Centre, we are moving one step closer to our ultimate goal of developing a cure for CF,” said William Skach, MD, executive vice president and chief scientific officer of the CF Foundation.

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CF is caused by mutations in the CFTR gene, leading to dysfunction of the CFTR protein encoded by this gene. Recently, a class of therapies called CFTR modulators has become widely available. These therapies work by correcting specific defects in the CFTR protein and they have proven to be highly effective at treating patients with specific mutations. However, they are of benefit only in people with those specific mutations.

“We are all incredibly excited about the huge effect that CFTR modifiers like Kaftrio [Trikafta in the U.S.] are likely to have for many people with CF. However, we have been and will continue to be absolutely committed to funding innovative and world-leading research that will have the same life-changing impact for all people with cystic fibrosis,” Allen said.

Conceptually, the aim of gene editing is to “correct” the mutation in the DNA of a person’s cells. The technique being studied in the new SRC is called CRISPR (clustered regularly interspaced short palindromic repeats).

The CRISPR system was developed based on a naturally occurring system that bacteria use to fight off infectious viruses. In bacteria, the system works by recognizing the sequences of viral DNA and destroying it.

In the context of gene editing, CRISPR basically works by “cutting out” the specific piece of DNA that has the mutation. Then, researchers can prompt the cell’s normal repair machinery to correct the cut, but this time with no mutation.

In principle, this technique can be applied to correct all CF-causing mutations.

The scientists who led the development of the CRISPR method were awarded the Nobel Prize for Chemistry in 2020.

In the SRC, researchers will be testing different variations on the CRISPR system to determine which one(s) might be best-suited for therapeutic use in CF. They also will be testing different ways of delivering the gene-editing system to cells in the body, including a strategy in which cells are edited outside the body and then inserted back into the patient.

“Our new SRC focuses on the key questions that we need to answer to bring gene editing therapies closer to the clinic. We have developed a range of tools and gained a lot of experience in applying gene editing in CF, now we need to refine these and work out the best way to proceed towards therapies for cystic fibrosis,” said Stephen Hart, PhD, a professor at University College London.

“It’s a very exciting area of science to work in and our thanks go to the Cystic Fibrosis Trust and the CF Foundation, and their supporters for the opportunity to continue this research,” Hart added.


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