Explores motor neuron diseases, discussing their classification, pathological signatures, etiology, and potential treatments, with a focus on Spinal Muscular Atrophy.
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 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.
Explores Alzheimer's disease, anxiety, pain, and the renin-angiotensin system, including the development of the first renin inhibitor for clinical treatment.
