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Gene therapy (What are disease that can be cured using gene therapy?…
Gene therapy
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What is gene therapy?
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US Food and Drugs Administration(FDA): Products that mediate their effects by transcription and/ or translation of transferred genetic material into host genome using nuceli acid, viruses, or genetically engineered microorganism.
EMA: products that contain active substances of recombinant nuclei acid administered into human beings with the purpose to regulate, repairs, replace, adding, or deleting a genetic sequence.
Future planning on gene therapy:
Able to allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery
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Introduction of gene therapy (based on journal)
Def:
The transfer of genetic material to cure a disease or at least to improve the clinical status of a patient
Basic concept:
Transform viruses into genetic shuttles, which would deliver the gene of interest into the target cell
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