Gene Therapy

The use of CRISPR/Cas9 gene editing improves the efficacy of ultrasound cancer therapy. Dec. 8, 2021 — Sonodynamic therapy employs ultrasound in conjunction with drugs to cause harmful reactive oxygen species (ROS) to be released at the site of a tumor. Gene therapy has the potential to treat a wide variety of diseases, including cancer, cystic fibrosis, heart disease, diabetes, haemophilia, and AIDS. Researchers are still investigating when and how to use gene therapy. Gene therapy in Pharmaceutical Science is currently only available in the United States as part of a clinical trial. By 2020, the remission rate for ALL patients treated with Kymriah was expected to be around 85%. After a year, more than half had no relapses. Novartis intends to track the outcomes of all patients who received the therapy for the next 15 years in order to better understand how long it will be effective.

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