CF
What is Cystic Fibrosis?
What is a genetic disorder that mainly affects the lungs and digestive system
What does CRISPR allow scientists to do?
What is Edit genes by cutting and repairing DNA
How many new CF cases are diagnosed each year in the U.S.?
What is approximately 1,000
What models have been used and shown successful in vivo CFTR correction.
What is Animal models (PIGS)
What gene is mutated in Cystic Fibrosis?
What is the CFTR gene
What is the name of the protein CRISPR uses to cut DNA?
What is Cas9
What age are most people diagnosed with CF
What is Before age 2
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What does the CFTR protein normally do?
What is it transports chloride ions in and out of cells
What is the guide RNA used for in CRISPR?
What is to lead Cas9 to the exact spot in the DNA to cut
What percentage of CF cases are diagnosed by age 2?
What is Over 75%
What are some side effects that researchers are seeing during CRISPR experimentation?
Chemicals/Electricity would hurt and kill human lung cells so they cant be used in patients
What happens when the CFTR protein doesn’t work?
What is thick mucus builds up, blocking airways and causing infections
How can CRISPR be used to treat CF?
What is By cutting and fixing the CFTR mutation in cells.
What are the chances two carriers will have a child with CF?
What is 1 in 4
What are lipid nanoparticles (LNPs) being used for in CRISPR therapy?
What is delivering CRISPR tools safely into the lungs?
What are three systems in the body affected by CF?
What is Respiratory, digestive, reproductive
What’s one major challenge scientists face when using CRISPR in real patients?
What is Safely delivering it to lung cells in people with thick mucus
What is one reason early diagnosis is important for CF?
What is It helps start treatments sooner to manage symptoms and prevent complications
In addition to lung improvement, what other health benefit has gene therapy shown in some studies?
What is increased lean body mass or improved body composition?