How Can Gene Therapy be used to Prevent SCD (Sickle Cell Disease)?
What is Sickle Cell Disease?
The sickle cell disease is a genetic red blood cell disorder caused by a mutation in hemoglobin. This mutation causes the usual hemoglobin to become less flexible and stiff, turning into a sickle shape. This can cause problems because red blood cells need to be flexible and transportable to carry oxygen and flow through big and small vessels. The sickle shaped cells however are too large and sticky causing blood clots and slow down the blood flow. Meaning often, not enough oxygen is able to reach tissues. This causes extreme pain and possible damage to the organs. While regular red blood cells survive 90 to 120 days, sickle cells survive a small …show more content…
The fast dieng rate of the cells makes the body work extremely hard because he always needs to reproduce new ones. This often results into the body not having enough cells causing anemia - a disease where one does not have enough energy. SCD is known to be the most common blood inherited disorder in the united states affecting 70’000 to 80’000 people. (nih.gov 2017) The life expectancy for this disease varies from person to person, however the common average now is 40 to 60 years old. The only cure so far for this disease is a hematopoietic stem cell transplantation (HSCT). However, the problem is that many people with sickle cell disease are too old for transplantation or do not have a good enough genetic matched donor. Gene therapy should be the solution in which, no matter what age or genes, …show more content…
Everyone is able to get gene therapy, just like everyone is able to get a virus. Another benefit is that the cure is not harmful at all since it is a natural reaction. The therapy uses a virus which is not harmful to humans. The suppression of the mutated cell happens without our awareness. The biggest advantage and the reason why gene therapy could be used more often is because it makes tissues and organs work properly again. Unlike many pharmaceuticals which may hinder the symptoms and signs that may come with a disease, however but can not cure the actual
What is Sickle Cell Disease?
The sickle cell disease is a genetic red blood cell disorder caused by a mutation in hemoglobin. This mutation causes the usual hemoglobin to become less flexible and stiff, turning into a sickle shape. This can cause problems because red blood cells need to be flexible and transportable to carry oxygen and flow through big and small vessels. The sickle shaped cells however are too large and sticky causing blood clots and slow down the blood flow. Meaning often, not enough oxygen is able to reach tissues. This causes extreme pain and possible damage to the organs. While regular red blood cells survive 90 to 120 days, sickle cells survive a small …show more content…
The fast dieng rate of the cells makes the body work extremely hard because he always needs to reproduce new ones. This often results into the body not having enough cells causing anemia - a disease where one does not have enough energy. SCD is known to be the most common blood inherited disorder in the united states affecting 70’000 to 80’000 people. (nih.gov 2017) The life expectancy for this disease varies from person to person, however the common average now is 40 to 60 years old. The only cure so far for this disease is a hematopoietic stem cell transplantation (HSCT). However, the problem is that many people with sickle cell disease are too old for transplantation or do not have a good enough genetic matched donor. Gene therapy should be the solution in which, no matter what age or genes, …show more content…
Everyone is able to get gene therapy, just like everyone is able to get a virus. Another benefit is that the cure is not harmful at all since it is a natural reaction. The therapy uses a virus which is not harmful to humans. The suppression of the mutated cell happens without our awareness. The biggest advantage and the reason why gene therapy could be used more often is because it makes tissues and organs work properly again. Unlike many pharmaceuticals which may hinder the symptoms and signs that may come with a disease, however but can not cure the actual