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In vivo gene therapy is a type of gene therapy that involves the direct delivery of genetic material into the body to treat a specific disease or condition. This is done by introducing therapeutic genes into the patient's cells, either by using viral vectors or non-viral methods. One example of in vivo gene therapy is the treatment of inherited retinal diseases, such as Leber congenital amaurosis (LCA). In this case, a functional copy of the defective gene responsible for the disease is introduced into the patient's retinal cells. This can be achieved by using a viral vector, such as an adeno-associated virus (AAV), to deliver the therapeutic gene to the target cells in the retina. Once inside the cells, the therapeutic gene produces the functional protein that is lacking in patients with LCA, thereby improving their vision. Please note that I am unable to access specific pages or figures from a book. If you need more detailed information, I recommend referring to the specific pages mentioned in your question or consulting reliable sources on the topic.
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