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3 ημέρες 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.
Gene therapy approaches to replace a faulty gene with a healthy gene have been proposed and are being studied for treating some genetic diseases. As of 2017, 11.1% of gene therapy clinical trials targeted monogenic diseases.
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...
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...
11 Σεπ 2021 · The term gene therapy refers to the use of different nucleic acids (DNA or RNA) as therapeutic molecules to treat different diseases. It relies on the use of nucleic acid molecules to add a specific gene to a diseased cell (gene augmentation) or to inhibit a specific gene in a diseased cell (gene inhibition).
26 Οκτ 2021 · Gene therapies use various strategies to supply cells with healthy genes, influence gene activity or tweak the genome directly.
23 Απρ 2024 · Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease. It holds promise as a treatment for a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
