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.
Explores the development and challenges of antibody-based therapeutics, including chimeric and humanized antibodies, alternative scaffolds, and in vitro selection.
