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The direct delivery of the therapeutic gene (DNA) into the target cells of a particular tissue of a patient constitutes in vivo gene therapy (Fig. 13.6). Many tissues are the potential candidates for this approach. These include liver, muscle, skin, spleen, lung, brain and blood cells.
1 ημέρα πριν · Gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis and cancer.
1 Νοε 2021 · Thanks to that new knowledge and steadfast investigations, gene therapy researchers can now point to a growing list of successful gene therapies. Here are four of the most promising. Some...
23 Μαρ 2023 · Gene therapy can involve inserting genes into an individual's cells and tissues to treat a disease. This diagram shows an example of ex vivo gene therapy. (Image credit: Aldona...
16 Νοε 2020 · Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation...
8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...
