Symdeko for treating cystic fibrosis

The U.S. Food and Drug Administration (FDA) has approved Symdeko (ivacaftor/tezacaftor tablets and ivacaftor tablets) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor.

Do not take Symdeko if you take certain medicines or herbal supplements such as: antibiotics such as rifampin (Rifamate, Rifater) or rifabutin (Mycobutin) seizure medicines such as phenobarbital, carbamazepine (Tegretol, Carbatrol, Equetro), or phenytoin (Dilantin, Phenytek)

Symdeko consists of 2 different tablets. The yellow tablet is marked with ‘V100’ and contains the medicines tezacaftor and ivacaftor. Take one yellow tablet in the morning. The light blue tablet is marked with ‘V150’ and contains the medicine ivacaftor. Take one light blue tablet in the evening. Take the yellow tablet and the light blue tablet about 12 hours apart.

Always take Symdeko with food that contains fat. Foods like egg, butter, peanut butter, cheese, pizza, and whole-milk dairy products such as whole milk, cheese, and yogurt. If you miss a dose and it is six hours or less from the time you usually take the yellow tablet in the morning or the light blue tablet in the evening, take the missed dose with food that contains fat as soon as you can. Then take your next dose at your usual time and it is more than six hours from the time you usually take the yellow tablet in the morning or the light blue tablet in the evening, do not take the missed dose. Take your next dose at the usual time with food that contains fat. Do not take more than usual dose to make up for a missed dose.

Symdeko can cause dizziness in some people who take it. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how Symdeko affects you. Avoid food or drink that contains grapefruit or Seville oranges while you are taking Symdeko. It can cause serious side effects, including: high liver enzymes in the blood.

Call your doctor right away if you have any of the following symptoms of liver problems:pain or discomfort in the upper right stomach (abdominal) area, yellowing of your skin or the white part of your eyes, loss of appetite, nausea or vomiting, dark amber-colored urine. Abnormality of the eye lens (cataract) in some children and adolescents treated with Symdeko or treated with ivacaftor alone. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment to look for cataracts. The most common side effects include: headache, nausea, sinus congestion and dizziness.
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DNA and RNA editing could heal many diseases

Scientists have discovered two gene editing techniques to fix mutations that cause diseases like cystic fibrosis and Duchenne muscular dystrophy. Both diseases, and about half all human genetic disorders, are caused by mutations in single letters in the human genome, in which an 'A' appears where there should be a 'B.'

The newly-developed gene editing systems can target the smallest units of human DNA or RNA to undo the mutation that causes cystic fibrosis. One system edits DNA in the genome itself, while the other targets RNA, which transports genetic messages for making proteins. The editing systems work in living cells, and if researchers can find ways to deliver them to human patients safely and effectively, they could be used to reverse the mutations that cause genetic diseases.

DNA and RNA contain four base components: adenine, thymine, guanine and cytosine. Cystic fibrosis is caused by an inherited genetic mutation that leads to abnormal mucus production in the lungs and digestive system. The thicker-than-normal mucus builds up in and blocks airways. It can be managed with breathing machines, inhalers and medications, but some affected by it will eventually need lung transplants. There is no cure for cystic fibrosis and it can be fatal.

Cystic fibrosis could be prevented or corrected if only there were a 'G' in the genome where the disease's victims have an 'A.' The new gene editing technologies could rewrite the part of the genome or its messenger that spells cystic fibrosis. The gene editing system is technically called the Adenine Base Editor, or ABE.

The ‘A’ in ABE is for ‘adenine,’ one of four chemical bases that are the smallest elements of our genomes. Adenine is always paired with thymine, and guanine is always paired with cytosine. ABE targets the ‘A,’ adenine, and rearranges its atoms to turn it into guanine. So, where there is an incorrect AT set of base pairs in the genome, ABE can reset it to a GC.

These genetic editors give scientists the remarkable ability to rewrite any mutated base pair in the genome. The gene editors are developments on the CRISPR technology which allows scientists to efficiently target and edit the genome.

RNA editing avoids interfering with the genome itself. Because RNA plays a communication role in humans, rather than being the fundamental genetic information itself, changes to it might be more flexible, and reversible.
However, RNA degrades over time, so the impermanence of changes to its component parts (called nucleoside bases) could be disadvantageous too.
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E-cigarette can cause lung diseases

E-cigarettes lead to as many lung diseases as tobacco products,
researchers compared saliva samples from tobacco smokers, e-cigarette smokers and nonsmokers. They found that e-cigarette smokers were likely to develop dangerous proteins associated with lung diseases such as COPD and cystic fibrosis and that the devices are not better for people than regular cigarettes.

E-cigarettes might not be the ideal alternative smokers addicted to tobacco are looking for. Previous research has proven that e-cigarettes can cause lifelong damage to the heart, one puff of an e-cigarette is all it takes to increase the risk of having a heart attack. E-cigarette smokers have elevated levels of neutrophil-extracellular-trap NET related proteins in their airways. NET proteins fight off pathogens, but increased levels of them can lead to inflammatory lung illnesses.

E-cigarettes are as dangerous as smoking - just ONE puff could be all it takes to increase the risk of a heart attack. E-cigarettes do inflict life-long damage on nonsmokers' hearts that is similar to tobacco cigarettes. Replacing tobacco products with e-cigarettes is dangerous, some of the flavoring agents and other products used in e-cigarettes are toxic.

The proteins are associated with COPD and cystic fibrosis, both of which make it hard for patients to breathe. E-cigarette smokers also have increased NET levels outside of their lungs, according to the study. This can cause cell death in tissues that line organs and blood vessels. E-cigarette smokers have an increased risk of suffering from bronchitis, asthma, bronchiectasis and wheezing. E-cigarettes is as bad as tobacco cigarettes
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