Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.
Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.
Delves into instrumental variable approaches for optimal treatment regimes, including precision medicine and personalized recommendations, with a focus on identification conditions and robust classification-based estimators.
