Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.
Delves into the molecular basis of genetic diseases, discussing specific examples like Phenylketonuria and Haemophilia A, and the development of small molecule drugs for genetic disorders.
Explores the genetic basis of Prader-Villy syndrome and its comparison with Angelman syndrome, emphasizing DNA methylation patterns and chromosomal abnormalities.
