Monday, September 28, 2020

Cystic Fibrosis Gene Therapy


Genetic diseases can be very serious, and even life threatening. Unfortunately, with most genetic diseases, we simply do not have the science available as of yet to prevent those diseases from passing along family lines. Cystic Fibrosis is one such genetic disease, but there is hope in the form of cystic fibrosis gene therapy.

What is Cystic Fibrosis?

Cystic Fibrosis is a disease which is inherited through the genes, and overall, it has a negative effect on the whole body. Most people who have Cystic Fibrosis, also known as CF and as Sixty Five Roses to children who cannot properly announce the name of the disease, will suffer progressive disability. Early death is also a part of CF.

When a person has CF, they have cysts in the pancreas, and often have difficulty breathing due to infections in the lungs. These are the two most serious effects of the disease. Other symptoms include frequent sinus infections, diarrhea, slow growth, and infertility.

What is Gene Therapy?

To understand gene therapy for cystic fibrosis, you must first consider what gene therapy is overall. For gene therapy, genes are actually injected into the person’s tissues. There are two types of gene therapy. The first is Germ Line Gene Therapy and the second is Somatic Gene Therapy. For Cystic Fibrosis it is Somatic Gene Therapy.

What are the Latest Cystic Fibrosis Gene Therapy Developments?

In March of 1999, it was reported that gene therapy had the ability to improve one of the problems associated with cystic fibrosis. Fortunately, this was not the first breakthrough in this field, or the last.

Gene therapy first came into discussion in 1972, when it was first mentioned in a medical journal. Gene therapy was not approved in the United States, however, until 1990. In 2002, it was widely reported that there were dangers to this type of treatment, when some patients developed health problems that resembled leukemia. Despite this, the treatment was still used and improvements were made.

Unfortunately, in the case of cystic fibrosis, gene therapy may only be a temporary solution to a lifelong problem. It doesn’t always work, and when it does work, the results are not long term. The same is true, thus far, for all other health problems that gene therapy is used for.

It is also important to note that gene therapy works best for conditions that are caused by mutation of a single gene, as opposed to problems that are caused by the mutation of multiple genes, which is the case for cystic fibrosis.

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Medically trained in the UK. Writes on the subjects of injuries, healthcare and medicine. Contact me

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