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The era of gene therapy

Typically, therapeutic gene therapies involve the transfer of genetic material into cells to reverse an abnormal condition or induce a new trait. Depending on the underlying genetic problem, various strategies such as addition, edition (repair), and deletion/knockout (inactivation) could be used. Sometimes gene therapy is used to add a normal and functional copy of an allele to increase gene expression, such as adding a normal human clotting factor IX allele to produce enough factor IX in hemophilia type B. Also, gene transferred by vectors...

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