U.S. FDA approves first treatment based on gene-editing technology

La FDA de Estados Unidos aprueba el primer tratamiento basado en la tecnología de edición genética

The FDA, also known as the U.S. Food and Drug Administration, has approved the use of gene editing technology to treat sickle cell disease. These are two treatments that are being approved for the first time and are a novelty in the field of technology and medical science.

Casgevy and Lyfgenia, a gene editing breakthrough that proposes to improve the quality of life of patients suffering from sickle cell anemia

With this approval, the FDA intends to help improve the health of patients suffering from this genetic disorder. In this type of anemia, the red blood cells are transformed from a flattened disk structure to a crescent structure. They also become stiffer and stickier, something that affects patients‘ overall health.

Normally, someone who suffers from this ailment is more exposed to seriousness in infections, being more likely to suffer complications in them or to recover later. They are people who should avoid temperature contrasts, crowded environments in times of great virulence or extreme fatigue.

A breakthrough for gene therapy

The FDA has classified this good news as “a groundbreaking advance” in the gene editing and gene therapy sector. Casgevy works by genome editing, something that has not been seen before, creating a major revolution and attention from medical professionals.

Currently, in the United States alone, it has been estimated that sickle cell disease affects about 100,000 people. It is more common in African Americans and less common in Hispanic Americans. The disease leads to a mutation of hemoglobin, which renders it less able to properly transport oxygen to all tissues of the body.

Although it is not a serious ailment, the sufferer is likely to feel more fatigue than someone who does not suffer from it. In addition, this type of anemia can cause severe pain and damage to various organs of the human body. Something that affects the survival of the person, and can lead to premature death if there is a misdiagnosis or inadequate treatment.

The President of the United States has already stated his opinion

Joe Biden has expressed his opinion on what has happened, commenting that it is something “tremendously promising”. He alluded to how important it is to develop additional life-saving treatments, bringing hope to millions of Americans living with “rare diseases.”

In his statement released by the White House, he also commented that this new breakthrough for medicine and gene editing also “represents the power of medical innovation” seeking to improve the lives of hundreds of American citizens.

The president was kind enough to explain what it is like for a sickle cell anemia patient to live in a normal way, just as another healthy person might. He recalled that many of them have to take breaks from their work due to the discomfort that chronic pain causes in their lives, generating “serious health complications” due to the obstacles they suffer.

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