However, these medications are only effective in people with specific CFTR mutations. Therefore, doctors will choose the type of CTFR modulator depending on the specific mutation someone has.

In CF, gene editing corrects the disease-causing mutation in the CFTR gene, resulting in a potentially permanent fix to the CFTR gene. Unlike other types of genetic therapies, a gene editing ...

This is particularly effective for patients with specific gating mutations that prevent the CFTR protein from opening properly. Correctors: Many CF mutations cause the CFTR protein to misfold.

Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue ...

While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation.

While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation.

ALYFTREK also offers a potentially transformative option for approximately 150 people with CF in the U.S. with one of 31 mutations who are now eligible for a CFTR modulator for the first time.

There are more than 2,000 known mutations in the CFTR gene, which lead to different levels of the severity of the disease. Lentiviral vectors are a type of gene therapy that exploit the ability of ...

While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation.