Gene Editing to Cure Cystic Fibrosis?
Cystic fibrosis (CF) is a genetic disorder that mostly affects the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term symptoms include breathing difficulties and coughing up mucus as a result of repeated lung infections. A sweat test and genetic tests diagnose the disorder. One gets this disease when individuals with CF inherit two copies of the defective CF gene — one copy per parent. All parents should have at least one copy of the defective gene. Individuals with only one copy of the defective CF gene are considered carriers, but the disorder is not present in them. However, there might finally be a cure for CF, it is a new emerging technology called gene editing — specifically CRISPR.
How can CRISPR help?
CRISPR technology is a basic but potent tool for genome editing. This makes it easy for researchers to change DNA sequences and adjust gene function. The Cas9 or in other terms, “CRISPR-associated” protein which is also an enzyme that behaves like a pair of molecular scissors, capable of cutting through regular DNA strands. CRISPR is a type of gene-editing technology allowing scientists to simply cut and paste genes into DNA more quickly and accurately. It is based on a targeted system of defense that destroys DNA originally found in some prokaryotes — which is a unicellular organism that does not contain a nucleus nor a membrane. CRISPR uses RNA, a molecule capable of reading genetic information, and CRISPR uses a specific RNA to find a particular part of DNA and sends out an enzyme called Cas9, this is the enzyme capable of editing the genes.
The major pros of CRISPR are that it is affordable and there is an “undo” option present. You can remove a mistake you have made or even a minute flaw in your genetic code, you could even delete a more complicated gene altogether and it is super simple as well. There are also some new CRISPR techniques that can change a single letter in the DNA. CRISPR is a new exponential technology and has the power to cure numerous diseases that do not have a solution.
How can we cure Cystic Fibrosis?
People with CF only live up to 40 years old. However, CRISPR can expand that lifespan by actually curing the disease.
Researchers have shown that CRISPR can be used in human lung cells originating from cystic fibrosis patients to repair the most common mutation behind the disease. The next move is to study it in humans, something that both Editas Medicine and CRISPR Therapeutics intend to do. However, multiple different mutations in the CFTR gene can cause cystic fibrosis, meaning different CRISPR therapies will need to be developed for different genetic defects. Editas said it will look at the most common mutations, as well as some of the unusual mutations for which no cure is available.
Why hasn’t this been done yet!?
Well, if I must be frank, we do not have enough knowledge about our human genes and how they work so, we can’t go right ahead and start changing up our DNA sequence. There are numerous genes in our bodies, and we haven’t even studied them all so if there is one mistake — we could end up tweaking one vital part of our bodies that is needed for survival.
But, scientists are constantly working and studying, to figure and learn how each gene works so we can cure various diseases like cancer, lupus, blindness, and more in the future!
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