Sickle-cell disease is a good candidate for studying genetic disease because ; to see if gene therapy can improve survival and prevent symptoms and complications associated with sickle cell disease, as well as to weigh the risks of gene therapy against the potential long-term benefits for sickle cell patients.
What is Genetic Disease?
Genetic diseases, also known as genetic disorders, are caused by changes in DNA, which are known as genetic mutations. Changes in the instructions for making a protein are caused by genetic mutations. Genetic mutations cause changes in the instructions for making a protein. These changes can prevent or alter protein function, as well as prevent protein production entirely. Genetic mutations are mostly inherited from our parents, which means that one or both of your parents passed them down to you.
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