Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.
Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.
Discusses approaches in gene therapy for different diseases and the advantages of gene transfer over conventional drugs, as well as viral vectors for gene delivery.
Covers the immune system's defense mechanisms against pathogens, including physical barriers, innate immunity, immune cell functions, and the complement system.
