How Successful Is Gene Therapy In Curing Genetic Disorders?

In this essay, a case study based on Cystic Fibrosis will be looked at and how successful gene therapy is in curing cystic fibrosis and evaluate the advantages and disadvantages.

Introduction: What is Cystic Fibrosis?

Cystic fibrosis is an inherited disease. It causes some glands in the body not to work properly. This condition causes the buildup of sticky mucus in the lungs, and digestive system, but also the pancreas, liver kidney and intestine. It is caused by the presence of a mutation in both copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. People with a single working copy are carriers and are normally healthy however, when cystic fibrosis transmembrane conductance regulator is not working properly, secretions which are normally thin instead become thick and sticky. With time this causes a lot of infections, and other health issues. (Ricco Santiago)

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The gene which encodes the CFTR was first cloned in 1989, encodes a 1480 amino acid containing protein. The cftr forms a chloride permeable membrane which is regulated by phosphorylation and is found on a variety of tissues to varying degrees. (Laboratory medicine, 2003 online)

In the United States, about 800 to 900 people are diagnosed with cystic fibrosis each year. There are about 30,000 people in the United States who are currently suffering from this disease. The number of people with CF is rising each year as more people are diagnosed early and live much longer now than they did 30 years ago. (Cunningham, M. D, and Taussig, M.D. CF Foundation, n.d. Online)

There are two types of gene therapy

• Somatic therapy – this involves altering the alleles in the body cells, particularly the cells that are most affected by the disorder. For example, cystic fibrosis (CF) is a genetic disorder that is very damaging to the respiratory system, so somatic therapy for CF targets the epithelial cells lining the lungs. Somatic therapy doesn’t affect the individual`s sex cells (sperm or egg) though, so any offspring could inherit the disease.
• Germ line therapy – this also involves altering the alleles in the sex cell. This means that every cell of any offspring produced from these cells will be affected by the gene therapy and they won`t inherit the disease. Germ line therapy in human is currently illegal though. (A level Biology OCRA, page 224, by Richard Parsons, 2018)

There is no cure for cystic fibrosis now, however gene therapy helps the patients to manage the disease better and now and live much longer,

Case Studies: Gene therapy holds the promise to transform medicine and create other options for patients who are living with difficult, and even incurable, diseases. Since 2017 three causes of gene therapy products has been approved in the United States by the Food and Drug Administrations. Two of them involving reprogram of a patient's own cells to attack a deadly cancer cells, and also the most recent approved one was a product targets a disease caused by mutations a specific gene. (FDA, n.d. online)

Gene therapy involves altering alleles inside a cell to cure genetic disorders

How you do this depends on whether the faulty gene is caused by a dominate allele or two recessive alleles. If it is caused by two recessive alleles you can add a working dominant allele to make up for them. However, if the genetic disorder is caused by a dominant allele you can silence the dominant allele by sticking a bit of DNA in the middle of the allele, so it does not work anymore.

With any medical researches, there are advantages, and disadvantages.

Advantages of gene therapy include: It could prolong the lives of people with genetic disorders and could give people with genetic disorders a better quality of life. Also, Carriers of genetic disorders might be able to convince a baby without that disorder. It may decrease the number of people that suffer from genetic disorders (only in Germ line therapy.

Disadvantages of gene therapy include: The technology could potentially be used in ways other than for medical treatment, such as for treating the cosmetic effects of ageing. There is the potential to do more harm than good by using the technology. And there is concern that gene therapy is expensive, some people believe that health service resource could be better spend on other treatments that have passed clinical trials. (Parson, 2018)

To have cystic fibrosis a child must inherit a copy of the faulty gene from each parent. The parents may not have the disease themselves, however they must be a carrier for the child to have it. Cystic fibrosis is an awful condition and tends to get worse over time and can be deadly if left untreated and can lead to serious infection or the lungs may stop working as they should which could cause other medical issues. Due to the advances of technology and medical research pee with cystic fibrosis (CF) are now living much happier life and longer. They can live up to 40 years of age now, whereas they only lived up to 10 years of age in 1960s.

Gene therapy holds the promise to transform medicine and create other options for patients who are living with difficult, and even incurable, diseases. Since 2017 three causes of gene therapy products has been approved in the United States by the Food and Drug Administrations. Two of them involving reprogram of a patient's own cells to attack a deadly cancer cells, and the most recent approved was a product targets a disease caused by mutations a specific gene. (FDA, n.d. online)

The concept of gene therapy seems straightforward; however, this is clearly an oversimplification, and scientist have identified risk factors related to gene therapy using viral vectors. Viruses can usually infect more than one type of cell. when viral vectors are used to carry genes into the body, they might infect healthy cells as well as causing cancer. And the long-term side effects are still unknown. Another danger is that the new gene might be inserted in the wrong location in the DNA and cause harmful mutations of other cells. (Gene Therapy Net, n.d. online)

Referencing

1. Available at https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work (Accessed 25/10/2020 Available at https://www.nhs.uk/conditions/cystic-fibrosis/ (Accessed 25/10/2020)
2. https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work
3. A-Level Biology OCR A (2019) Gene Therapy Revision, page 224, by Richard Parsons, 2018) https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/what-causes-cystic-fibrosis (accessed 19/10/2020)
4. Cystic Fibrosis Foundation (https://www.cff.org/PDF-Archive/An-Introduction-to-Cystic-Fibrosis-for-Patients-and-Their-Families/ (James C. Cunningham, M.D. Lynn M. Taussig, M.D. (accessed 07/11/2020) Available at (https://www.genetherapynet.com/viral-vector/risk-factors.html accessed 07/11/2020)