How does gene therapy work?
A mutation leads to missing or malformed proteins can either be harmless or can lead to serious diseases. In case mutation is harmful and leads to serious diseases, doctors use gene therapy as a treatment. Lab technicians isolate normal DNA and package it into a vector, which acts as a molecular delivery truck. These vectors are composed of viral DNA sequences. They either injected or given intravenously (by IV) into a specific tissue in the patient’s body. Thenceforth, the target cell (cell where a mutation occurred) is infected with the vector. After the infection takes place, the vector unloads its DNA cargo fostering the cell to produce proper proteins and restoring it to normal. Moreover, lab technicians are optimizing viral vectors as well as developing non-viral vectors (liposomes & naked DNA) that have fewer unexpected side effects. In 1985, Drs. W. French Anderson and Michael Blaese accomplished the first successful human …show more content…
The somatic gene therapy treats the faulty genes and replaces them with the correct genes. By doing so, the body will produce the right proteins needed to eliminate the diseases causing gene. However, this type of therapy cannot be passed down. While the germline gene therapy passes down the corrected gene to next generations. This procedure inserts the corrected gene into the reproductive cells, so that it can be inherited. Therefore, genetic disorder will not be inherited. Moreover, almost all genetic diseases are curable with gene therapy. For example, hemophilia is a genetic disorder in which missing proteins help blood to form clots. Patients diagnosed with hemophilia can lose large amounts of blood through internal bleeding and even minor cuts. In 2011, Sebastian Misztal was a patient suffering from hemophilia. However, after his liver cells received an adeno-associated viral vector that enclosed the gene, he no longer had spontaneous bleeding
A mutation leads to missing or malformed proteins can either be harmless or can lead to serious diseases. In case mutation is harmful and leads to serious diseases, doctors use gene therapy as a treatment. Lab technicians isolate normal DNA and package it into a vector, which acts as a molecular delivery truck. These vectors are composed of viral DNA sequences. They either injected or given intravenously (by IV) into a specific tissue in the patient’s body. Thenceforth, the target cell (cell where a mutation occurred) is infected with the vector. After the infection takes place, the vector unloads its DNA cargo fostering the cell to produce proper proteins and restoring it to normal. Moreover, lab technicians are optimizing viral vectors as well as developing non-viral vectors (liposomes & naked DNA) that have fewer unexpected side effects. In 1985, Drs. W. French Anderson and Michael Blaese accomplished the first successful human …show more content…
The somatic gene therapy treats the faulty genes and replaces them with the correct genes. By doing so, the body will produce the right proteins needed to eliminate the diseases causing gene. However, this type of therapy cannot be passed down. While the germline gene therapy passes down the corrected gene to next generations. This procedure inserts the corrected gene into the reproductive cells, so that it can be inherited. Therefore, genetic disorder will not be inherited. Moreover, almost all genetic diseases are curable with gene therapy. For example, hemophilia is a genetic disorder in which missing proteins help blood to form clots. Patients diagnosed with hemophilia can lose large amounts of blood through internal bleeding and even minor cuts. In 2011, Sebastian Misztal was a patient suffering from hemophilia. However, after his liver cells received an adeno-associated viral vector that enclosed the gene, he no longer had spontaneous bleeding