Huntington’s disease is a severe inherited neurodegenerative disorder lacking effective treatments. A UCL research team has pioneered AMT-130, a gene therapy showing promise in slowing disease progression. Early trials indicate safety and significant clinical benefits, marking a hopeful shift towards potentially transformative therapies for Huntington’s and similar conditions.
Tag: gene therapy
Cancer treatment has evolved with CAR T-cell therapy, where patients’ immune cells are engineered to target cancer. Developed by UCL’s Professor Waseem Qasim, it has shown promise in reversing aggressive blood cancers, with a patient cancer-free for three years. However, it faces challenges in treating solid tumors and manufacturing costs.
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