This lecture explores the properties of commonly used viral vectors, such as adenoviruses, adeno-associated viruses, retroviruses, and lentiviruses, highlighting their advantages and limitations for gene therapy applications.
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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.
