Gene Therapy in Hemoglobinopathy Hemoglobinonpathy is the lack of a molecule or total blood hemoglobin chains. Hemoglobin is found in red blood cells. Example of the disease is alpha-thalassemia. This happens due to lack of one or two series of alpha-Zhlobin or beta (where the shortfall in one or two Zhlobin beta), for example, inheritance thalassemia is inherited through a recessive trait autosomal recessive physical. There are differences between the alpha thalassemia and beta thalassemia. Alpha symptoms are usually immediately after birth no need time for the appearance of the period and the second is that the alpha chain of hemoglobin is more important and vital than the beta chain. If there is lack in total red blood cell - such
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First trial was in 1990 on four years old girl has inherited disorder that cause damage of her immune system. First death case occurred at 1999 for 18-yeares old male after suffering of sever immune reaction as a result of using viral vector to deliver the genetic unit into patient cell. Gene therapy is the procedure that involve transferring what we can call it corrected genetic unit into patient cell doing in lab for treating genetic disorder by using special genetic tools. We can use gene therapy technique to treat gene disorder occurring as a result of: (i)Infectious agent: including viruses or bacteria, (ii)Cancer: uncontrolled cell proliferation, (iii) Immune disease: including autoimmune disease and hypersensitivity, (iiii) Inherited disorders: as hemogobinopathy for example sickle cell disease and different types of thalassemia. There are two types of gene therapy; Classical gene therapy and Non-classical gene therapy. The strategies of gene therapy is: Increase gene product, Killing affected cell- like cancer cells- Correction the mutation, and stopping specific gene expression: specially to fix the disorder occurring as a result of inherited or infectious agent. The general mechanism of this is blocking specific gene expression that cause the disease. There are three types of vector – which is used to deliver the corrected gene to patient cell to achieve the aim of gene …show more content…
Development tool team to deliver the correct gene.A viral vector called GLOBE can then be modified cells genetically engineered with the Globe to restore hemoglobin production before being re-run again in the patient 's body through intravenous injection. The focus of this work is important, not only to show that we can restore hemoglobin production in human cells, but this genetic transfer-based approach does not impair the biological features of the cells and is associated with any significant risk for the human genome corrected.
Ethical issue of gene therapy was a major challenge since the experiment start. There are many point should be considered when established a new gene therapy trials like other new medication. Gene therapy classified as dangerous potential due to transferring the therapeutic gene as part of whole process so, the safety of the patient should be guarantee. The main problem coming from three thing; nucleic acid, delivery system, and the procedure
First trial was in 1990 on four years old girl has inherited disorder that cause damage of her immune system. First death case occurred at 1999 for 18-yeares old male after suffering of sever immune reaction as a result of using viral vector to deliver the genetic unit into patient cell. Gene therapy is the procedure that involve transferring what we can call it corrected genetic unit into patient cell doing in lab for treating genetic disorder by using special genetic tools. We can use gene therapy technique to treat gene disorder occurring as a result of: (i)Infectious agent: including viruses or bacteria, (ii)Cancer: uncontrolled cell proliferation, (iii) Immune disease: including autoimmune disease and hypersensitivity, (iiii) Inherited disorders: as hemogobinopathy for example sickle cell disease and different types of thalassemia. There are two types of gene therapy; Classical gene therapy and Non-classical gene therapy. The strategies of gene therapy is: Increase gene product, Killing affected cell- like cancer cells- Correction the mutation, and stopping specific gene expression: specially to fix the disorder occurring as a result of inherited or infectious agent. The general mechanism of this is blocking specific gene expression that cause the disease. There are three types of vector – which is used to deliver the corrected gene to patient cell to achieve the aim of gene …show more content…
Development tool team to deliver the correct gene.A viral vector called GLOBE can then be modified cells genetically engineered with the Globe to restore hemoglobin production before being re-run again in the patient 's body through intravenous injection. The focus of this work is important, not only to show that we can restore hemoglobin production in human cells, but this genetic transfer-based approach does not impair the biological features of the cells and is associated with any significant risk for the human genome corrected.
Ethical issue of gene therapy was a major challenge since the experiment start. There are many point should be considered when established a new gene therapy trials like other new medication. Gene therapy classified as dangerous potential due to transferring the therapeutic gene as part of whole process so, the safety of the patient should be guarantee. The main problem coming from three thing; nucleic acid, delivery system, and the procedure