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18 Οκτ 2022 · Gene therapy is at the forefront of modern medicine. By making precise changes to the human genome, these sophisticated technologies can potentially lead to one-time lifelong cures for infectious and non-communicable diseases (e.g. HIV, sickle cell disease) that affect tens of millions of people around the globe, most of whom live in LMICs.
13 Αυγ 2024 · Gene therapy is a relatively new treatment designed to alleviate disease by modifying defective genes or altering the production of proteins by faulty genes. There are several ways that healthy genes can be inserted into the body, such as inside a deactivated virus or inside a fat particle.
21 Ιαν 2023 · Gene therapy can treat certain genetic conditions by editing diseased cells. Learn about its uses, safety, and potential downsides here.
24 Μαρ 2022 · Benefits. In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders, such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. They also may be used to treat cancers or infections, including HIV.
Gene therapy based on viral vectors utilizes the natural ability of viruses to deliver genetic material to cells, and a large part of research has been devoted toward generating novel, more efficient, and safer delivery tools employing gammaretroviruses, lentiviruses, adenoviruses, and adeno-associated viruses.
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 technologies....
4 Ιουλ 2019 · To date, therapies that alter the genes responsible for disease have been showing success in clinical trials. My goal is to streamline the regulatory approval of gene therapies with early patient access and subsequent data.