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Everything You Need to Know About the Gene Therapy for Genetic-Related Diseases

What most people don’t know is that the genes that are actually found in the cells of a body of a living thing, plays a huge part when it comes to making sure that the body is healthy and is functioning that right way.

Then again, if there is a defective gene that is mixed with the normal ones, it could definitely affect the way the body would function, in short, it could actually make the person ill. So because of this, medical researchers have been doing everything they can to basically find a way to be able to modify defective genes or to replace those defective ones with the ones that are healthy so that they could prevent people from getting ill and they have actually been doing this for more than ten years now. Finally, this 2017, the U.S. Food and Drug Administration has finally approved certain gene therapy products, continue reading to learn more.


It is a great year for scientists and researchers from the medical field indeed because, after decades of studying and looking for ways to be able to modify and fix those messed up genes that cause diseases, the U.S. Food and Drug Administration has finally approved three gene therapy products. These products have been approved to treat people who are diagnosed with some kind of an inherited vision loss or blindness.

Luxturna, which is a brand new gene therapy, is said to have the power to treat children as well as adults who have unfortunately inherited a type of vision loss that could actually lead to them being permanently blind. This will be the very first time the U.S. Food and Drug Administration has actually approved such kind of treatment especially when it comes to illnesses that are caused by genetic mutations. Luxturna is actually by Spark Therapeutics and this treatment is said to be able to help people who are suffering from Leber congenital amaurosis, which is basically a kind of a genetic retinal disease that is considered to be rare. This treatment is also for the patients who happen to have the RPE65 mutated gene that is actually very important when it comes to making a specific enzyme that is essential to normalize a person’s vision because a defective one could do the opposite wherein it could make the persona permanently and completely blind.


According to Spark Therapeutic, Luxturna actually uses a modified virus to be able to make a healthier copy of a gene that is identical to a person’s retinal cell that would prevent them from losing vision with the help of eye surgery. This would then make the non-defective copy cell to replace the broken one and it could help with the production of the enzymes that are needed by the body to be able to have the person’s vision normal again.

In a statement released by the commissioner of the Food and Drug Administration Scott Gottlieb on their official website, it is indeed a historical day when it comes to the gene therapy field since this is the first time this has been approved especially with how the gene therapy is used as well as how it can expand. This milestone will definitely be very helpful especially when it comes to researching for more uses for different kinds of challenging diseases.

FDA’s approval of the Luxturna is actually expected by the industry since its shares went up to about 6.8 percent. This specific medical research field has been struggling to actually make a gene therapy that would pass the incredibly high standards of the FDA especially when it comes to treating diseases such as sickle-cell anemia as well as hemophilia and of course other genetic related diseases.

However, people are already on how much these treatments are going to cost, but according to Sparks Therapeutics, they wouldn’t announce the prices until January of 2018 but experts and analysts think that it could cost a million for both eyes. This is, of course, a lot of when for a procedure that wouldn’t guarantee a perfect vision.


It took scientists and researchers decades to get the Luxturna to be approved, but how exactly does gene therapy work? According to scientists, gene therapy basically replaces defective genes from the body but it would always depend on the problem. They could also add genes to prevent certain diseases and turn off those that are the actually causing the problems. The Luxturna went into a lot of process such as being tested for its effectiveness and most especially if it is safe to be used on the human body.

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