Cystic Fibrosis Foundation Therapeutics Inc. made its first major award last month to advance gene editing research in cystic fibrosis. Editas Medicine Inc. will explore the potential of using what some scientists refer to as “a tiny pair of scissors” to snip out the genetic defect that causes CF.
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The Cystic Fibrosis Foundation is funding research into gene editing techniques to see if they can be used to fix the mutations that cause cystic fibrosis. One of the most popular techniques is CRISPR-Cas9. To see how this might work for CF, watch this video.
I felt unrelenting hope watching the first plenary of this year’s North American Cystic Fibrosis Conference. As I learned about progress that has been made in sickle cell disease, and how those learnings may help us develop a genetic therapy for CF, it showed me that the CF community is supporting people like me who can’t take modulators.
La fibrosis quística (FQ) es una enfermedad genética. Escuche las presentaciones de los expertos, el Doctor Fadel Ruiz, director del centro de fibrosis quística de Baylor y el Doctor Carlos Milla, director del centro de fibrosis quística de Stanford y sus respuestas a las principales preguntas de la comunidad sobre terapias genéticas para la FQ. El panel fue moderado por Sylvia Mazuera, madre de dos hijos, el menor de los cuales tiene FQ.
Back in 2015, my genetic sequencing revealed one nonsense mutation. But recently, I learned I have another nonsense mutation that may not have been identified when my genes were first sequenced. As more mutation-dependent therapies are developed, it’s crucial that people with CF have accurate information so they can make informed decisions.
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.
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.