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The goal of gene therapy is to correct an undesirable trait or disease by introducing a modified copy of a gene into a target cell. In most cases, the purpose is not to replace a defective gene in the host cell but rather to provide a new copy so that the correct protein can be expressed and the detrimental effects of the defective gene neutralized. While technically, any genetic disorder may be treated by gene therapy, there are some limitations. First, the precise genetic mechanism of the... więcej
Magill’s Medical Guide (Online Edition), 2023. 3p.
